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 ...

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 ...

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 ...

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 ...

- DYNE-101 in DM1: Dyne plans to initiate global Registrational Expansion Cohort of ACHIEVE trial with registrational dose of 6.8 mg/kg Q8W following study data showing splicing correction and robust ...

(RTTNews) - Dyne Therapeutics, Inc. (DYN), Tuesday announced that the U.S. Food and Drug Administration has granted Fast Track designation for DYNE-101 for the treatment of myotonic dystrophy type 1, ...

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 ...

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 ...

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 ...

The $12.5 billion acquisition of Avidity Biosciences Inc. by Novartis AG strengthens the company’s neuroscience pipeline and marks the second biggest deal that’s been announced this year. It also is ...