Using brain organoids, researchers discover mutational commonalities between muscular dystrophy type 1 and Rett syndrome, suggesting the potential of a similar treatment for both. Myotonic dystrophy ...

AOC 1001 consists of a proprietary monoclonal antibody that binds to transferrin receptor 1 conjugated with a small interfering RNA. The Food and Drug Administration (FDA) has granted Fast Track ...

Please provide your email address to receive an email when new articles are posted on . The FDA has granted breakthrough therapy designation to a proprietary oligonucleotide conjugate for the ...

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 ...

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 ...

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 ...

University of Virginia School of Medicine researchers have identified new insights and potential treatment approaches for muscle loss in myotonic dystrophy type 1 (DM1), the most common form of ...

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 ...

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 ...