2f:I[703058,["/_next/static/immutable/chunks/3bhq_5eh1d760.js","/_next/static/immutable/chunks/2iy5wruuedsh6.js","/_next/static/immutable/chunks/2pfsjpn6k6zlg.js","/_next/static/immutable/chunks/26c7v3_3uwnwu.js","/_next/static/immutable/chunks/00yc7rcvua6a7.js","/_next/static/immutable/chunks/37ktb71aq3480.js","/_next/static/immutable/chunks/2d50b-7eu4558.js","/_next/static/immutable/chunks/06_yt270h41rg.js","/_next/static/immutable/chunks/2d6noqpp6ehas.js","/_next/static/immutable/chunks/1sx186c93bpzg.js","/_next/static/immutable/chunks/3dc-1n866s65i.js","/_next/static/immutable/chunks/20x2y96ldu5wx.js","/_next/static/immutable/chunks/2w1bvggzb0ga8.js","/_next/static/immutable/chunks/2051ffx3xzio9.js"],"PaperListPageClient"] 30:T425,Overall, 552 participants with wild-type ATTR-CM (mean [SD] age, 78 [6.3] years; 92.0% male and 8.0% female) and 59 participants with variant ATTR-CM (mean [SD] age, 73 [7.7] years; 77.3% male and 22.7% female) were randomized (mITT population), including 35 with p.Val142Ile. Consistent efficacy was observed in wild-type and variant subgroups for ACM/CVH through month 30 and ACM through month 42. At month 30, acoramidis reduced the risk of ACM/first CVH vs placebo by 31% in ATTRwt-CM (hazard ratio [HR], 0.69; 95% CI, 0.52-0.90; P = .007) and by 59% in ATTRv-CM (HR, 0.41; 95% CI, 0.21-0.81; P = .01). ACM was reduced through month 42 with HRs of 0.70 (95% CI, 0.50-0.98; P = .04) and 0.41 (95% CI, 0.19-0.93; P = .03) in the ATTRwt-CM and ATTRv-CM groups, respectively. Consistent treatment benefit was observed in participants with ATTRwt-CM and ATTRv-CM for secondary end points. Within variant subgroups (p.Val142Ile vs non-p.Val142Ile), consistent treatment benefits were observed for ACM/CVH through month 30 and ACM through month 42.31:Taf4,Transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive disease caused by misfolded transthyretin (TTR), occurs as wild-type (ATTRwt-CM) or variant (ATTRv-CM) forms. p.Val142Ile is the most common variant in the US, linked to rapid progression and increased mortality. Acoramidis achieves near-complete (≥90%) TTR stabilization and showed clinical benefit in the 30-month ATTRibute-CM trial and through month 42 in the ongoing open-label extension (OLE). To evaluate the efficacy of acoramidis in ATTRwt-CM, ATTRv-CM, and variant subgroups (p.Val142Ile and non-p.Val142Ile). This international, multicenter, phase 3, randomized placebo-controlled study took place from April 2019 to May 2023 with ongoing OLE (month 42). ATTRibute-CM enrolled 632 participants with ATTR-CM; 611 of 632 were included in the modified intention-to-treat (mITT) population. There were 380 participants who continued into the OLE. These data were analyzed from January 2025 to July 2025. Oral acoramidis, 712 mg, or placebo twice daily for 30 months, followed by 12 months of open-label treatment. All-cause mortality (ACM), cardiovascular-related hospitalizations (CVH), serum TTR, 6-minute walk distance, Kansas City Cardiomyopathy Questionnaire Overall Summary score, and N-terminal pro B-type natriuretic peptide in participants with ATTRwt-CM and ATTRv-CM. Post-hoc analyses were conducted in variant subgroups, including p.Val142Ile. Overall, 552 participants with wild-type ATTR-CM (mean [SD] age, 78 [6.3] years; 92.0% male and 8.0% female) and 59 participants with variant ATTR-CM (mean [SD] age, 73 [7.7] years; 77.3% male and 22.7% female) were randomized (mITT population), including 35 with p.Val142Ile. Consistent efficacy was observed in wild-type and variant subgroups for ACM/CVH through month 30 and ACM through month 42. At month 30, acoramidis reduced the risk of ACM/first CVH vs placebo by 31% in ATTRwt-CM (hazard ratio [HR], 0.69; 95% CI, 0.52-0.90; P = .007) and by 59% in ATTRv-CM (HR, 0.41; 95% CI, 0.21-0.81; P = .01). ACM was reduced through month 42 with HRs of 0.70 (95% CI, 0.50-0.98; P = .04) and 0.41 (95% CI, 0.19-0.93; P = .03) in the ATTRwt-CM and ATTRv-CM groups, respectively. Consistent treatment benefit was observed in participants with ATTRwt-CM and ATTRv-CM for secondary end points. Within variant subgroups (p.Val142Ile vs non-p.Val142Ile), consistent treatment benefits were observed for ACM/CVH through month 30 and ACM through month 42. The beneficial effect of acoramidis was observed consistently in ATTRwt-CM and ATTRv-CM groups. These hypothesis-generating results indicate that further studies are warranted to better characterize the therapeutic benefit of acoramidis in variant subgroups. ClinicalTrials.gov Identifiers: NCT03860935; NCT04988386.32:T1311, Importance Transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive disease caused by misfolded transthyretin (TTR), occurs as wild-type (ATTRwt-CM) or variant (ATTRv-CM) forms. p.Val142Ile is the most common variant in the US, linked to rapid progression and increased mortality. Acoramidis achieves near-complete (≥90%) TTR stabilization and showed clinical benefit in the 30-month ATTRibute-CM trial and through month 42 in the ongoing open-label extension (OLE). Objective To evaluate the efficacy of acoramidis in ATTRwt-CM, ATTRv-CM, and variant subgroups (p.Val142Ile and non-p.Val142Ile). Design, Setting, and Participants This international, multicenter, phase 3, randomized placebo-controlled study took place from April 2019 to May 2023 with ongoing OLE (month 42). ATTRibute-CM enrolled 632 participants with ATTR-CM; 611 of 632 were included in the modified intention-to-treat (mITT) population. There were 380 participants who continued into the OLE. These data were analyzed from January 2025 to July 2025. Interventions Oral acoramidis, 712 mg, or placebo twice daily for 30 months, followed by 12 months of open-label treatment. Main Outcomes and Measures All-cause mortality (ACM), cardiovascular-related hospitalizations (CVH), serum TTR, 6-minute walk distance, Kansas City Cardiomyopathy Questionnaire Overall Summary score, and N-terminal pro B-type natriuretic peptide in participants with ATTRwt-CM and ATTRv-CM. Post-hoc analyses were conducted in variant subgroups, including p.Val142Ile. Results Overall, 552 participants with wild-type ATTR-CM (mean [SD] age, 78 [6.3] years; 92.0% male and 8.0% female) and 59 participants with variant ATTR-CM (mean [SD] age, 73 [7.7] years; 77.3% male and 22.7% female) were randomized (mITT population), including 35 with p.Val142Ile. Consistent efficacy was observed in wild-type and variant subgroups for ACM/CVH through month 30 and ACM through month 42. At month 30, acoramidis reduced the risk of ACM/first CVH vs placebo by 31% in ATTRwt-CM (hazard ratio [HR], 0.69; 95% CI, 0.52-0.90; P = .007) and by 59% in ATTRv-CM (HR, 0.41; 95% CI, 0.21-0.81; P = .01). ACM was reduced through month 42 with HRs of 0.70 (95% CI, 0.50-0.98; P = .04) and 0.41 (95% CI, 0.19-0.93; P = .03) in the ATTRwt-CM and ATTRv-CM groups, respectively. Consistent treatment benefit was observed in participants with ATTRwt-CM and ATTRv-CM for secondary end points. Within variant subgroups (p.Val142Ile vs non-p.Val142Ile), consistent treatment benefits were observed for ACM/CVH through month 30 and ACM through month 42. Conclusions and Relevance The beneficial effect of acoramidis was observed consistently in ATTRwt-CM and ATTRv-CM groups. These hypothesis-generating results indicate that further studies are warranted to better characterize the therapeutic benefit of acoramidis in variant subgroups. Trial Registration ClinicalTrials.gov Identifiers: NCT03860935 ; NCT04988386 25:[[["$","script","0",{"type":"application/ld+json","dangerouslySetInnerHTML":{"__html":"{\"@context\":\"https://schema.org\",\"@type\":\"CollectionPage\",\"name\":\"acoramidis: generic Research & Clinical Takeaways\",\"description\":\"Explore peer-reviewed papers on acoramidis (generic). Clinician-written summaries and actionable practice insights. Updated daily.\",\"url\":\"https://www.reachrx.ai/hub/tag/generic/acoramidis\",\"breadcrumb\":{\"@type\":\"BreadcrumbList\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https://www.reachrx.ai\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"Research Hub\",\"item\":\"https://www.reachrx.ai/hub\"},{\"@type\":\"ListItem\",\"position\":3,\"name\":\"Generics\",\"item\":\"https://www.reachrx.ai/hub/tag/generic\"},{\"@type\":\"ListItem\",\"position\":4,\"name\":\"acoramidis\",\"item\":\"https://www.reachrx.ai/hub/tag/generic/acoramidis\"}]}}"}}]],["$","$L2f",null,{"title":"acoramidis: Research & Clinical Takeaways","description":"Explore research on acoramidis (generic) — efficacy, safety, and comparative data.","papers":[{"doi":"10.1001/jamacardio.2026.3803","pmid":"42671012","title":"Prognostic Thresholds of Tricuspid Regurgitation in Transthyretin Amyloid Cardiomyopathy","journal_title":"JAMA Cardiology","journal_issn":"2380-6583","published_at":"2026-08-31T00:00:00.000Z","url":"https://doi.org/10.1001/jamacardio.2026.3803","source_score":27,"is_retracted":false,"is_open_access":true,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-09-10T06:19:58.311Z","updated_at":"2026-09-10T06:19:58.311Z","tags":[{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"age_group","value":"older_adult","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"brand","value":"Vyndamax","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"category","value":"Cardiac & Cardiovascular Systems","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"condition","value":"Transthyretin Amyloid Cardiomyopathy","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"condition","value":"Tricuspid Regurgitation","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"generic","value":"acoramidis","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"generic","value":"patisiran","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"generic","value":"tafamidis","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"generic","value":"vutrisiran","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"impact_flag","value":"practice_changing","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"level_of_evidence","value":"2","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"modality","value":"Echocardiography","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"sample_size","value":"n=1124","value_numeric":1124},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"sex","value":"mixed","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"study_design","value":"Multicenter Prospective Cohort Study","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"warning","value":"Tafamidis brand tag 'Vyndamax' applied as the most common drug in this cohort (95.5% of ATTR-specific therapy); acoramidis brand name (BridgeBio's 'Attruby') was not tagged as a brand due to very low use (n=2) and uncertainty about approval status at time of data collection.","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"warning","value":"The cohort is predominantly older adult males (median age 80, 76.9% male) from European tertiary centers, which may limit generalizability to younger, female, or non-European ATTR-CM populations.","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.3803","tag_type":"warning","value":"The 'practice_changing' flag is applied because this framework reclassifies a substantially larger proportion of ATTR-CM patients as high/extreme risk compared to all current guidelines; however, prospective interventional validation (e.g., whether reclassification changes treatment decisions or outcomes) has not yet been demonstrated.","value_numeric":null}],"summaries":{"what_studied":"This international multicenter cohort study (8 centers across Austria, Italy, Germany, and the Netherlands; January 2016–February 2026) derived and validated disease-specific echocardiographic TR severity thresholds in 1,124 newly diagnosed ATTR-CM patients, comparing their prognostic performance for all-cause mortality and heart failure hospitalization (HFH) against current ESC/EACTS, AHA/ACC, ASE, and TVARC grading schemes.","key_result":"Spline-derived thresholds classified 29.7% of patients (n=334) as high or extreme risk, versus only 11.6% (n=130) with guideline-defined severe TR (P<0.001). The new framework carried the highest adjusted hazard ratios for mortality (HR 1.41; 95% CI 1.23–1.62) and HFH (HR 1.31; 95% CI 1.12–1.54), with extreme-risk patients showing 4- to 6-fold higher mortality vs. low-risk. Time-dependent AUC was consistently superior to ESC/EACTS–AHA/ACC at all time points; in the non-amyloid HF cohort (n=364), the framework offered no discriminatory advantage.","limitations":"- Derivation and validation cohorts differed in several baseline characteristics (e.g., ATTR subtype distribution, ATTR-specific therapy use, SGLT-2i use), which may limit generalizability.\n- Missing quantitative TR data required median imputation by local TR grade, though a complete-case sensitivity analysis was performed.\n- The atrial functional TR subgroup was defined by a pragmatic proxy (right atrial dilatation + normal RV size) rather than a validated dedicated algorithm.","practice_takeaway":"Standard guideline TR grading misses nearly two-thirds of ATTR-CM patients who carry significant prognostic risk; clinicians should consider applying quantitative thresholds (VCW ≥3 mm, EROA ≥0.15 cm², RegVol ≥10 mL for intermediate risk) specific to ATTR-CM's restrictive low-flow physiology. These disease-specific cut-offs should not be applied to non-amyloid heart failure populations, where conventional schemes perform better."}},{"doi":"10.1001/jamacardio.2026.2413","pmid":"42525404","title":"Effect of Acoramidis on Heart Failure–Related Health Status","journal_title":"JAMA Cardiology","journal_issn":"2380-6583","published_at":"2026-07-29T00:00:00.000Z","url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC13420175/","source_score":21,"is_retracted":false,"is_open_access":true,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-08-06T13:21:59.826Z","updated_at":"2026-08-06T13:21:59.826Z","tags":[{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"age_group","value":"older_adult","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"category","value":"Cardiac & Cardiovascular Systems","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"condition","value":"Heart Failure","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"condition","value":"Transthyretin Amyloid Cardiomyopathy","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"generic","value":"acoramidis","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"impact_flag","value":"practice_changing","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"level_of_evidence","value":"2","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"modality","value":"Transthyretin Stabilizer","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"sample_size","value":"n=611","value_numeric":611},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"sex","value":"mixed","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"study_design","value":"Randomized Controlled Trial","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"warning","value":"Full text was not accessible; summary is based solely on the abstract and results sections provided.","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"warning","value":"Level of evidence tagged as '2' because this is a secondary/post hoc analysis of a phase 3 RCT rather than a primary pre-registered outcome analysis.","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.2413","tag_type":"warning","value":"The KCCQ-OS analysis at month 30 is a pre-specified secondary outcome, but the 'alive and not worse,' 'alive and well,' and 'alive and better' composite endpoints are post hoc — interpret those NNT values with some caution.","value_numeric":null}],"summaries":{"what_studied":"This secondary analysis of the ATTRibute-CM phase 3 RCT evaluated the effect of acoramidis 800 mg twice daily vs placebo on HF-related health status (KCCQ-OS score) in 611 adults with ATTR-CM over 30 months.","key_result":"Acoramidis produced a statistically significant, clinically meaningful improvement in KCCQ-OS vs placebo (LSM difference +9.9 points; 95% CI, 6.0–13.9; P < .001). At 30 months, 47% of acoramidis recipients were \"alive and not worse\" vs 30% on placebo (NNT = 6), and 46% were \"alive and well\" vs 31% (NNT = 7).","limitations":"- This is a secondary/post hoc analysis of the trial, so some comparisons (\"alive and better\") were not pre-specified.\n- The population was predominantly older (mean age 77.2 years) and male (90.8%), limiting generalizability to women and younger patients.\n- KCCQ data were only available in surviving patients at each time point, which may introduce survivorship bias given the mortality difference between arms.","practice_takeaway":"In ATTR-CM, acoramidis not only reduces mortality and hospitalizations but also meaningfully preserves patient-reported quality of life — roughly 1 in 6 additional patients remain alive and not worse compared to placebo (NNT = 6). Clinicians can use these KCCQ data to counsel patients on expected functional benefits over a 2.5-year treatment course."}},{"doi":"10.1001/jamacardio.2026.0819","pmid":"41911489","title":"Long-Term Durability of Acoramidis Efficacy in Transthyretin Amyloid Cardiomyopathy","journal_title":"JAMA Cardiology","journal_issn":"2380-6583","published_at":"2026-03-30T00:00:00.000Z","url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC13036634/","source_score":114,"is_retracted":false,"is_open_access":true,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-05-18T13:25:18.543Z","updated_at":"2026-05-18T13:25:18.543Z","tags":[{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"age_group","value":"older_adult","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"brand","value":"Attruby","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"category","value":"Cardiac & Cardiovascular Systems","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"condition","value":"Transthyretin Amyloid Cardiomyopathy","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"generic","value":"acoramidis","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"impact_flag","value":"practice_changing","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"level_of_evidence","value":"2","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"modality","value":"Transthyretin Stabilizer","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"sample_size","value":"n=632","value_numeric":632},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"sex","value":"mixed","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"study_design","value":"Open-Label Extension of Randomized Controlled Trial","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"warning","value":"Level of evidence assigned as 2 (controlled trial with OLE extension) rather than 1, given the absence of a randomized comparator arm beyond month 30.","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"warning","value":"Only 389 of 632 original trial participants enrolled in the OLE; selective enrollment may introduce survivor bias and overestimate long-term benefit.","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"warning","value":"The full text PDF metadata appears to be from the FDA prescribing label for Attruby (acoramidis), not the full journal article — content beyond the abstract and results section is limited to label pages with no extractable body text. Summary is based solely on the abstract and results sections.","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"warning","value":"The OLE dose (800 mg BID) differs from the FDA-approved commercial dose (712 mg BID, as two 356 mg tablets); clinicians should use the approved labeling dose in practice.","value_numeric":null}],"summaries":{"what_studied":"This open-label extension (OLE) of the ATTRibute-CM RCT evaluated the long-term efficacy and safety of acoramidis 800 mg twice daily through month 54 in adults with ATTR-CM, comparing continuous acoramidis (n=263) vs. placebo-to-acoramidis switchers (n=126) who crossed over at month 30.","key_result":"Continuous acoramidis reduced all-cause mortality (HR 0.55; 95% CI 0.42–0.74; P<.001), cardiovascular mortality (HR 0.51; 95% CI 0.36–0.71; P<.001), and first cardiovascular hospitalization (HR 0.53; 95% CI 0.42–0.69; P<.001) through month 54, with stabilization of NT-proBNP, KCCQ-OS score, and 6MWD; late switchers showed stabilization of NT-proBNP and KCCQ-OS and improvement in sTTR and 6MWD, but did not fully recover the outcomes benefit seen with continuous treatment.","limitations":"- OLE design with no placebo arm beyond month 30 limits controlled comparison at later timepoints.\n- High dropout (389 of 632 enrolled in OLE), raising survivor bias concerns.\n- Population was predominantly older males (mean age 77.3 years, 90.2% male), limiting generalizability to women and younger patients.","practice_takeaway":"Start acoramidis early in ATTR-CM — 54-month data show sustained, incremental reductions in death and hospitalization with continuous therapy that are not fully recovered by switching later. Do not delay or interrupt treatment while awaiting disease progression."}},{"doi":"10.1001/jamacardio.2025.4477","pmid":"41205147","title":"Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy","journal_title":"JAMA Cardiology","journal_issn":"2380-6583","published_at":"2025-11-08T00:00:00.000Z","url":"https://doi.org/10.1001/jamacardio.2025.4477","source_score":103,"is_retracted":false,"is_open_access":true,"raw_metadata":{"ooir":{"day":"2026-01-16","doi":"10.1001/jamacardio.2025.4477","days":"14","issn":"2380-6583","rank":"5","field":"Clinical Medicine","score":"103","category":"Cardiac & Cardiovascular Systems","categories":["Cardiac & Cardiovascular Systems"],"_categories":["Cardiac & Cardiovascular Systems"]},"pubmed":{"doi":"10.1001/jamacardio.2025.4477","title":"Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: Results From ATTRibute-CM and Its Open-Label Extension.","journal":"JAMA cardiology","methods":"","results":"$30","abstract":"$31","keywords":[],"pubmed_id":"41205147","conclusions":"","publication_date":"2025-11-08T00:00:00"},"crossref":{"DOI":"10.1001/jamacardio.2025.4477","URL":"https://doi.org/10.1001/jamacardio.2025.4477","ISSN":["2380-6583"],"link":[{"URL":"https://jamanetwork.com/journals/jamacardiology/articlepdf/2841140/jamacardiology_alexander_2025_oi_250068_1767980815.15996.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"page":"57","type":"journal-article","issue":"1","score":1,"title":["Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy"],"author":[{"given":"Kevin M.","family":"Alexander","sequence":"first","affiliation":[{"name":"Stanford Amyloid Center, Division of Cardiovascular Medicine, Stanford University School of Medicine, Palo Alto, California"}]},{"given":"Margot K.","family":"Davis","sequence":"additional","affiliation":[{"name":"Division of Cardiology, University of British Columbia, Vancouver, British Columbia, Canada"}]},{"given":"Olakunle","family":"Akinboboye","sequence":"additional","affiliation":[{"name":"Laurelton Heart Specialists, Queens Heart Institute, Rosedale, New York, New York"}]},{"given":"John","family":"Berk","sequence":"additional","affiliation":[{"name":"Boston Medical Center, Boston University, Boston, Massachusetts"}]},{"given":"Kunal","family":"Bhatt","sequence":"additional","affiliation":[{"name":"Division of Cardiology of the Department of Medicine at Emory University, Emory Healthcare, Atlanta, Georgia"}]},{"given":"Francesco","family":"Cappelli","sequence":"additional","affiliation":[{"name":"Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence, Italy"}]},{"given":"Sarah A.M.","family":"Cuddy","sequence":"additional","affiliation":[{"name":"Heart and Vascular Center, Brigham and Women’s Hospital, Boston, Massachusetts"}]},{"given":"Marianna","family":"Fontana","sequence":"additional","affiliation":[{"name":"National Amyloidosis Centre, Division of Medicine, University College London, London, United Kingdom"}]},{"given":"Pablo","family":"Garcia-Pavia","sequence":"additional","affiliation":[{"name":"Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV, and Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid, Spain"}]},{"given":"Julian D.","family":"Gillmore","sequence":"additional","affiliation":[{"name":"Centre for Amyloidosis and Acute Phase Proteins, Division of Medicine, University College London, London, United Kingdom"}]},{"given":"Jan M.","family":"Griffin","sequence":"additional","affiliation":[{"name":"Division of Cardiology, Department of Medicine, Medical University of South Carolina, Charleston"}]},{"given":"Justin L.","family":"Grodin","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas"}]},{"given":"Daniel P.","family":"Judge","sequence":"additional","affiliation":[{"name":"Division of Cardiology, Department of Medicine, Medical University of South Carolina, Charleston"}]},{"given":"Michel G.","family":"Khouri","sequence":"additional","affiliation":[{"name":"Division of Cardiology, Department of Medicine, Duke University Health System, Durham, North Carolina"}]},{"given":"Kaitlyn","family":"Lam","sequence":"additional","affiliation":[{"name":"Western Australia Advanced Heart Failure and Cardiac Transplant Service, Fiona Stanley Hospital, Western Australia, Australia"}]},{"given":"Ahmad","family":"Masri","sequence":"additional","affiliation":[{"name":"Division of Cardiology, Oregon Health & Science University, Portland"}]},{"given":"Mathew S.","family":"Maurer","sequence":"additional","affiliation":[{"name":"Department of Medicine, Columbia University Irving Medical Center, New York, New York"}]},{"given":"Laura","family":"Obici","sequence":"additional","affiliation":[{"name":"Fondazione IRCCS Policlinico San Matteo, Pavia, Italy"}]},{"given":"Frederick L.","family":"Ruberg","sequence":"additional","affiliation":[{"name":"Boston University Chobanian & Avedisian School of Medicine, Boston Medical Center, Boston, Massachusetts"}]},{"given":"Nitasha","family":"Sarswat","sequence":"additional","affiliation":[{"name":"Division of Cardiovascular Medicine, University of Chicago Medicine, Chicago, Illinois"}]},{"given":"Keyur","family":"Shah","sequence":"additional","affiliation":[{"name":"Pauley Heart Center, Virginia Commonwealth University, Richmond"}]},{"given":"Prem","family":"Soman","sequence":"additional","affiliation":[{"name":"Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania"}]},{"given":"Lily","family":"Stern","sequence":"additional","affiliation":[{"name":"Department of Cardiology, Smidt Heart Institute, Cedars Sinai, Los Angeles, California"}]},{"given":"Richard","family":"Wright","sequence":"additional","affiliation":[{"name":"Pacific Heart Institute and Providence, St. John’s Health Center, Santa Monica, California"}]},{"given":"Kuangnan","family":"Xiong","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Xiaofan","family":"Cao","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Ted","family":"Lystig","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Jean-François","family":"Tamby","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Adam","family":"Castaño","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Leonid","family":"Katz","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Uma","family":"Sinha","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Jonathan C.","family":"Fox","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Scott D.","family":"Solomon","sequence":"additional","affiliation":[{"name":"Heart and Vascular Center, Brigham and Women’s Hospital, Boston, Massachusetts"}]},{"given":"Martha","family":"Grogan","sequence":"additional","affiliation":[{"name":"Cardiovascular Medicine, Mayo Clinic Rochester, Minnesota"}]}],"issued":{"date-parts":[[2026,1,1]]},"member":"10","prefix":"10.1001","source":"Crossref","volume":"11","created":{"date-time":"2025-11-08T16:30:17Z","timestamp":1762619417000,"date-parts":[[2025,11,8]]},"indexed":{"date-time":"2026-01-15T02:49:23Z","timestamp":1768445363652,"date-parts":[[2026,1,15]]},"subject":[],"abstract":"$32","language":"en","relation":{},"resource":{"primary":{"URL":"https://jamanetwork.com/journals/jamacardiology/fullarticle/2841140"}},"subtitle":["Results From ATTRibute-CM and Its Open-Label Extension"],"deposited":{"date-time":"2026-01-14T16:00:54Z","timestamp":1768406454000,"date-parts":[[2026,1,14]]},"issn-type":[{"type":"print","value":"2380-6583"}],"published":{"date-parts":[[2026,1,1]]},"publisher":"American Medical Association (AMA)","reference":[{"DOI":"10.1093/eurheartj/ehab072","key":"hoi250068r1","year":"2021","issue":"16","author":"Garcia-Pavia","volume":"42","first-page":"1554","unstructured":null,"article-title":"Diagnosis and treatment of cardiac amyloidosis: a position statement of the ESC Working Group on Myocardial and Pericardial Diseases.","journal-title":"Eur Heart J","doi-asserted-by":"publisher"},{"DOI":"10.1161/CIRCULATIONAHA.118.038169","key":"hoi250068r2","year":"2019","issue":"1","author":"Lane","volume":"140","first-page":"16","unstructured":null,"article-title":"Natural history, quality of life, and outcome in cardiac transthyretin amyloidosis.","journal-title":"Circulation","doi-asserted-by":"publisher"},{"DOI":"10.1016/j.jacc.2022.11.022","key":"hoi250068r3","year":"2023","issue":"11","author":"Kittleson","volume":"81","first-page":"1076","unstructured":null,"article-title":"2023 ACC expert consensus decision pathway on comprehensive multidisciplinary care for the patient with cardiac amyloidosis: a report of the American College of Cardiology Solution Set Oversight Committee.","journal-title":"J Am Coll Cardiol","doi-asserted-by":"publisher"},{"DOI":"10.1001/jama.2024.0442","key":"hoi250068r4","year":"2024","issue":"9","author":"Ruberg","volume":"331","first-page":"778","unstructured":null,"article-title":"Cardiac amyloidosis due to transthyretin protein: a review.","journal-title":"JAMA","doi-asserted-by":"publisher"},{"DOI":"10.1002/ejhf.2776","key":"hoi250068r5","year":"2023","issue":"4","author":"Porcari","volume":"25","first-page":"515","unstructured":null,"article-title":"Prevalence, characteristics and outcomes of older patients with hereditary versus wild-type transthyretin amyloid cardiomyopathy.","journal-title":"Eur J Heart Fail","doi-asserted-by":"publisher"},{"DOI":"10.1016/j.jchf.2020.09.011","key":"hoi250068r7","year":"2021","issue":"2","author":"Rapezzi","volume":"9","first-page":"115","unstructured":null,"article-title":"Efficacy of tafamidis in patients with hereditary and wild-type transthyretin amyloid cardiomyopathy: further analyses from ATTR-ACT.","journal-title":"JACC Heart Fail","doi-asserted-by":"publisher"},{"DOI":"10.1038/s41467-024-50231-1","key":"hoi250068r8","year":"2024","issue":"1","author":"Shetty","volume":"15","first-page":"6221","unstructured":null,"article-title":"Determinants of transthyretin levels and their association with adverse clinical outcomes among UK Biobank participants.","journal-title":"Nat Commun","doi-asserted-by":"publisher"},{"DOI":"10.1001/jamacardio.2020.5969","key":"hoi250068r9","year":"2021","issue":"3","author":"Greve","volume":"6","first-page":"258","unstructured":null,"article-title":"Association of low plasma transthyretin concentration with risk of heart failure in the general population.","journal-title":"JAMA Cardiol","doi-asserted-by":"publisher"},{"DOI":"10.1001/jamacardio.2024.4102","key":"hoi250068r10","year":"2025","issue":"2","author":"Christoffersen","volume":"10","first-page":"155","unstructured":null,"article-title":"Transthyretin tetramer destabilization and increased mortality in the general population.","journal-title":"JAMA Cardiol","doi-asserted-by":"publisher"},{"DOI":"10.1161/CIRCHEARTFAILURE.117.004000","key":"hoi250068r11","year":"2018","issue":"2","author":"Hanson","volume":"11","first-page":null,"unstructured":null,"article-title":"Use of serum transthyretin as a prognostic indicator and predictor of outcome in cardiac amyloid disease associated with wild-type transthyretin.","journal-title":"Circ Heart Fail","doi-asserted-by":"publisher"},{"DOI":"10.1016/j.jacc.2025.03.542","key":"hoi250068r12","year":"2025","issue":"20","author":"Maurer","volume":"85","first-page":"1911","unstructured":null,"article-title":"Early increase in serum transthyretin by acoramidis independently predicts improved survival in TTR amyloid cardiomyopathy.","journal-title":"J Am Coll Cardiol","doi-asserted-by":"publisher"},{"DOI":"10.1161/CIRCGEN.121.003356","key":"hoi250068r13","year":"2021","issue":"5","author":"Chandrashekar","volume":"14","first-page":null,"unstructured":null,"article-title":"Prevalence and outcomes of p.Val142Ile TTR amyloidosis cardiomyopathy: a systematic review.","journal-title":"Circ Genom Precis Med","doi-asserted-by":"publisher"},{"DOI":"10.1038/s41431-022-01235-2","key":"hoi250068r14","year":"2023","issue":"5","author":"Mazzarotto","volume":"31","first-page":"541","unstructured":null,"article-title":"Investigation on the high recurrence of the ATTRv-causing transthyretin variant Val142Ile in central Italy.","journal-title":"Eur J Hum Genet","doi-asserted-by":"publisher"},{"DOI":"10.1016/j.jmoldx.2013.08.001","key":"hoi250068r15","year":"2014","issue":"1","author":"Reddi","volume":"16","first-page":"68","unstructured":null,"article-title":"Homozygosity for the V122I mutation in transthyretin is associated with earlier onset of cardiac amyloidosis in the African American population in the seventh decade of life.","journal-title":"J Mol Diagn","doi-asserted-by":"publisher"},{"DOI":"10.1002/ejhf.3088","key":"hoi250068r16","year":"2024","issue":"2","author":"Razvi","volume":"26","first-page":"383","unstructured":null,"article-title":"Deep phenotyping of p.(V142I)-associated variant transthyretin amyloid cardiomyopathy: Distinct from wild-type transthyretin amyloidosis?","journal-title":"Eur J Heart Fail","doi-asserted-by":"publisher"},{"DOI":"10.3109/13506129.2015.1019610","key":"hoi250068r17","year":"2015","issue":"2","author":"Swiecicki","volume":"22","first-page":"123","unstructured":null,"article-title":"Hereditary ATTR amyloidosis: a single-institution experience with 266 patients.","journal-title":"Amyloid","doi-asserted-by":"publisher"},{"DOI":"10.1093/eurheartj/ehac544.1793","key":"hoi250068r18","year":"2022","issue":null,"author":"Razvi","volume":null,"first-page":null,"unstructured":null,"article-title":"A multi-modality, multi-parametric phenotyping study of transthyretin amyloid cardiomyopathy associated with the p.V142I TTR variant.","journal-title":"Eur Heart J","doi-asserted-by":"publisher"},{"DOI":"10.1021/acs.jmedchem.8b00817","key":"hoi250068r19","year":"2018","issue":"17","author":"Miller","volume":"61","first-page":"7862","unstructured":null,"article-title":"Enthalpy-driven stabilization of transthyretin by AG10 mimics a naturally occurring genetic variant that protects from transthyretin amyloidosis.","journal-title":"J Med Chem","doi-asserted-by":"publisher"},{"DOI":"10.1093/eurheartj/ehad655.989","key":"hoi250068r20","year":"2023","issue":null,"author":"Ji","volume":null,"first-page":null,"unstructured":null,"article-title":"Acoramidis produces near-complete TTR stabilization in blood samples from patients with variant transthyretin amyloidosis that is greater than that achieved with tafamidis.","journal-title":"Euro Heart J","doi-asserted-by":"publisher"},{"DOI":"10.1097/FJC.0000000000001726","key":"hoi250068r21","year":"2025","issue":"2","author":"Ji","volume":"86","first-page":"204","unstructured":null,"article-title":"Differential binding affinities and kinetics of transthyretin stabilizers.","journal-title":"J Cardiovasc Pharmacol","doi-asserted-by":"publisher"},{"DOI":"10.1056/NEJMoa2305434","key":"hoi250068r22","year":"2024","issue":"2","author":"Gillmore","volume":"390","first-page":"132","unstructured":null,"article-title":"Efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy.","journal-title":"N Engl J Med","doi-asserted-by":"crossref"},{"DOI":"10.1016/j.jacc.2024.11.042","key":"hoi250068r23","year":"2025","issue":"10","author":"Judge","volume":"85","first-page":"1003","unstructured":null,"article-title":"Efficacy of acoramidis on all-cause mortality and cardiovascular hospitalization in transthyretin amyloid cardiomyopathy.","journal-title":"J Am Coll Cardiol","doi-asserted-by":"crossref"},{"DOI":"10.1161/CIRCULATIONAHA.124.072771","key":"hoi250068r28","year":"2025","issue":"9","author":"Judge","volume":"151","first-page":"601","unstructured":null,"article-title":"Long-term efficacy and safety of acoramidis in ATTR-CM: initial report from the open-label extension of the ATTRibute-CM trial.","journal-title":"Circulation","doi-asserted-by":"crossref"},{"DOI":"10.1093/eurheartj/ehx589","key":"hoi250068r29","year":"2018","issue":"30","author":"Gillmore","volume":"39","first-page":"2799","unstructured":null,"article-title":"A new staging system for cardiac transthyretin amyloidosis.","journal-title":"Eur Heart J","doi-asserted-by":"crossref"},{"DOI":"10.1002/(SICI)1097-0258(19990615)18:11<1341::AID-SIM129>3.0.CO;2-7","key":"hoi250068r30","year":"1999","issue":"11","author":"Finkelstein","volume":"18","first-page":"1341","unstructured":null,"article-title":"Combining mortality and longitudinal measures in clinical trials.","journal-title":"Stat Med","doi-asserted-by":"crossref"},{"DOI":"10.1073/pnas.1300761110","key":"hoi250068r31","year":"2013","issue":"24","author":"Penchala","volume":"110","first-page":"9992","unstructured":null,"article-title":"AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretin.","journal-title":"Proc Natl Acad Sci U S A","doi-asserted-by":"crossref"},{"DOI":"10.1001/jama.2024.4467","key":"hoi250068r32","year":"2024","issue":"21","author":"Selvaraj","volume":"331","first-page":"1824","unstructured":null,"article-title":"Cardiovascular burden of the V142I transthyretin variant.","journal-title":"JAMA","doi-asserted-by":"crossref"},{"DOI":"10.1007/s11886-025-02220-z","key":"hoi250068r33","year":"2025","issue":"1","author":"Fahed","volume":"27","first-page":"66","unstructured":null,"article-title":"Race, genetics, and social determinants of health in transthyretin cardiac amyloidosis: a literature review and call to action.","journal-title":"Curr Cardiol Rep","doi-asserted-by":"crossref"},{"DOI":null,"key":"hoi250068r6","year":null,"issue":null,"author":null,"volume":null,"first-page":null,"unstructured":"Rowczenio? D, Wechalekar? A. Mutations in hereditary amyloidosis. Accessed October 21, 2025. http://www.amyloidosismutations.com","article-title":null,"journal-title":null,"doi-asserted-by":null},{"DOI":null,"key":"hoi250068r24","year":null,"issue":null,"author":null,"volume":null,"first-page":null,"unstructured":"European Medicines Agency. Beyonttra (acoramidis) [SmPC]. Accessed October 21, 2025. https://www.ema.europa.eu/en/medicines/human/EPAR/beyonttra","article-title":null,"journal-title":null,"doi-asserted-by":null},{"DOI":null,"key":"hoi250068r25","year":null,"issue":null,"author":null,"volume":null,"first-page":null,"unstructured":"BridgeBio Pharma Inc. ATTRUBY (acoramidis) [package insert]. Accessed October 21, 2025. https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/216540s000lbl.pdf","article-title":null,"journal-title":null,"doi-asserted-by":null},{"DOI":null,"key":"hoi250068r26","year":null,"issue":null,"author":null,"volume":null,"first-page":null,"unstructured":"GlobeNewswire. Beyonttr (acoramidis), the first near-complete TTR stabilizer (=90%), approved in Japan to treat ATTR-CM. Accessed October 21, 2025. https://www.globenewswire.com/news-release/2025/03/27/3050413/0/en/Beyonttra-acoramidis-the-First-Near-complete-TTR-Stabilizer-90-Approved-in-Japan-to-Treat-ATTR-CM.html","article-title":null,"journal-title":null,"doi-asserted-by":null},{"DOI":null,"key":"hoi250068r27","year":null,"issue":null,"author":null,"volume":null,"first-page":null,"unstructured":"Gov.uk. Press release: Acoramidis approved to treat wild-type or variant transthyretin amyloidosis in adults with cardiomyopathy. Accessed October 21, 2025. https://www.gov.uk/government/news/acoramidis-approved-to-treat-wild-type-or-variant-transthyretin-amyloidosis-in-adults-with-cardiomyopathy","article-title":null,"journal-title":null,"doi-asserted-by":null}],"short-title":[],"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,1,1]]}},"content-domain":{"domain":[],"crossmark-restriction":false},"original-title":[],"container-title":["JAMA Cardiology"],"published-print":{"date-parts":[[2026,1,1]]},"reference-count":33,"published-online":null,"references-count":33,"short-container-title":["JAMA Cardiol"],"is-referenced-by-count":0},"unpaywall":{"doi":"10.1001/jamacardio.2025.4477","is_oa":true,"title":"Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy","updated":"2025-12-10T03:52:38+00:00","published_date":"2025-11-08","best_oa_location":{"url":"https://jamanetwork.com/journals/jamacardiology/articlepdf/2841140/jamacardiology_alexander_2025_oi_250068_1762180369.48874.pdf","pmh_id":null,"url_for_pdf":"https://jamanetwork.com/journals/jamacardiology/articlepdf/2841140/jamacardiology_alexander_2025_oi_250068_1762180369.48874.pdf","url_for_landing_page":"https://doi.org/10.1001/jamacardio.2025.4477"}}},"created_at":"2026-01-18T22:40:12.285Z","updated_at":"2026-01-21T12:05:26.220Z","tags":[{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"age_group","value":"older_adult","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"category","value":"Cardiac & Cardiovascular Systems","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"condition","value":"Transthyretin Amyloid Cardiomyopathy","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"generic","value":"acoramidis","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"impact_flag","value":"confirms_prior","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"level_of_evidence","value":"2","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"modality","value":"Transthyretin Stabilizer","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"sample_size","value":"n=611","value_numeric":611},{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"sex","value":"mixed","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"study_design","value":"Randomized Controlled Trial","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"warning","value":"Absolute event counts and rates were not provided in the abstract.","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"warning","value":"Open‑label extension data inform the 42‑month mortality analysis and may introduce bias.","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2025.4477","tag_type":"warning","value":"Subgroup findings (variant and p.Val142Ile) are post hoc with small samples.","value_numeric":null}],"summaries":{"what_studied":"An international, multicenter, phase 3 randomized, placebo-controlled trial tested oral acoramidis 712 mg twice daily vs placebo for 30 months in transthyretin amyloid cardiomyopathy, with an open‑label extension to month 42 (mITT n=611; 552 wild‑type, 59 variant including 35 p.Val142Ile). Outcomes included all‑cause mortality (ACM), cardiovascular‑related hospitalizations (CVH), and key functional and biomarker measures.","key_result":"At month 30, acoramidis reduced ACM/first CVH vs placebo in ATTRwt-CM (HR 0.69; 95% CI 0.52-0.90; P=.007) and ATTRv-CM (HR 0.41; 95% CI 0.21-0.81; P=.01). Through month 42, ACM was lower with acoramidis in ATTRwt-CM (HR 0.70; 95% CI 0.50-0.98; P=.04) and ATTRv-CM (HR 0.41; 95% CI 0.19-0.93; P=.03), with consistent benefits across variant subgroups including p.Val142Ile.","limitations":"Variant subgroup and p.Val142Ile analyses were post hoc with small numbers (ATTRv n=59; p.Val142Ile n=35), limiting precision. Mortality results beyond 30 months derive from an open‑label extension, introducing potential bias.","practice_takeaway":"Acoramidis reduced death or first cardiovascular hospitalization at 30 months and reduced mortality through 42 months in both wild‑type and variant ATTR‑CM. Use across genotypes appears supported, while variant subgroup signals should be viewed as hypothesis‑generating."}}],"initialOffset":4,"fetchConfig":{"kind":"tags","tags":[{"tag_type":"generic","value":"acoramidis"}]},"breadcrumbItems":[{"name":"Home","href":"/"},{"name":"Research Hub","href":"/hub"},{"name":"Generics","href":"/hub/tag/generic"},{"name":"acoramidis"}],"hrefConfig":{"basePath":"/hub/papers","webViewQueryParams":{"hub_ctx":"tag","hub_tag_type":"generic","hub_tag_value":"acoramidis"}}}]]

Papers