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 ...

HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 -- 48-week trial will enroll approximately 150 ...

SAR446268 is a one-time adeno-associated viral gene therapy that delivers a vectorized RNA interference to silence DMPK expression. The Food and Drug Administration (FDA) has granted Fast Track ...

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 ...

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 ...

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 ...

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 ...

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 ...

DM1 primarily affects skeletal and cardiac muscle and is characterized by muscle weakness, difficulty breathing, and cognitive and behavioral impairment. The Food and Drug Administration (FDA) has ...

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 ...

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 ...