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Rare Disease Research

Scholars2
Papers2
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Related Papers (2)
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Effects of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults with Becker muscular dystrophy: results of a phase 1b, open-label study
errEBioMedicine
IF10.8
err2026-06-17
err0
errOAAI
errHan Phan; Ben Barthel; Molly Madden; Jeffrey A. Silverman; Abby Bronson; Elizabeth Thaler; Nicole Rempel Kilburn; Maria Amato; Monica Massaro; James MacDougall; Alan J. Russell; Joanne Donovan
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Dystrophinopathy in ACTION: The first 500 males enrolled in the Advanced Cardiac Therapies Improving Outcomes Network prospective dystrophinopathy registry
errNeuromuscular Disorders
IF2.8
err2025-11-08
err0
PREAI
errEmily A. Hayes; Chet R. Villa; Benjamin Kroslowitz; Deipanjan Nandi; Linda Cripe; Jonathan H. Soslow; Deepa Mokshagundam; Renata Shih; Bethany Wisotzkey; John J. Parent; Tyler Cunningham; Jennifer Conway; Paul Esteso; Brian F. Birnbaum; Svetlana B. Shugh; Frank J. Raucci; Beth D. Kaufman; Nelia Soares; Sonya Kirmani; Hugo R. Martinez; Katheryn Gambetta; Ashwin K. Lal; Carol A. Wittlieb-Weber
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errSave