Zeleciment basivarsen (z-basivarsen) demonstrated sustained functional improvement across multiple clinical measures in the ongoing ACHIEVE clinical trial - - Expect to complete enrollment in ACHIEVE ...
September 6, 2007 — A new study using a mouse model of myotonic dystrophy type 1 (DM1) has revealed an early step in the process by which a mutant DM gene leads to cardiac defects. Found in more than ...
Myotonic dystrophy type 1 incurs high healthcare costs, driven by age and comorbidities like cardiac and respiratory issues. Early detection and integrated management of multisystemic complications ...
Thurman M. Wheeler, MD, of the Department of Neurology at Massachusetts General Hospital, is the senior author of a paper published in Nature Communications, "Analysis of human urinary extracellular ...
People with Myotonic Dystrophy Type 1 (DM1), the most common adult-onset form of muscular dystrophy, progressively lose muscle mass and strength in their lower legs, hands, neck and face. The effects ...
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared progress in the Phase 1/2 multiple ascending dose (MAD) clinical study of SRP-1003 ...
Dyne Therapeutics announces FDA Fast Track designation for DYNE-101, targeting myotonic dystrophy type 1 with potential accelerated approval in 2026. Dyne Therapeutics has announced that its ...
Please provide your email address to receive an email when new articles are posted on . The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals ...
LONDON, Sept. 15, 2025 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held clinical-stage specialty biopharmaceutical company focusing on rare genetic disorders with limited or no ...
Myotonic Dystrophy Type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely affects multiple organs including skeletal muscle, heart, brain and the ...
June 19, 2008 — A new study confirms that adult patients with myotonic dystrophy type 1, the most common of the muscular dystrophies presenting in adults, are at high risk for arrhythmias and death ...
Researchers focused on Myotonic Dystrophy 1 heart problems are testing a novel approach to restore normal function. Myotonic Dystrophy Type 1 (DM1) is the most common adult-onset form of muscular ...
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