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 treatment, JUV-161, is Juvena’s lead candidate of engineered biologics for myopathies and metabolic diseases ...

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 ...

AOC 1001 continues to be generally well tolerated; Avidity provides more information on the rare serious adverse event in a single participant that led to the partial clinical hold AOC 1001 top-line ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Back to Healio A Southern California biopharmaceutical ...

WALTHAM, Mass., Oct. 06, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven ...

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 ...

(RTTNews) - Avidity Biosciences Inc. (RNA) announced new positive AOC 1001 data demonstrating improvement in multiple additional functional endpoints and favorable long-term safety and tolerability in ...

Myotonic dystrophy type 1 (DM1) is a rare, genetic disorder that causes progressive muscle weakness and wasting, with no currently approved medicines Paris, September 23, 2025. The US Food and Drug ...

- Patient-reported outcomes support clinical meaningfulness of improvements in function and strength - - Meaningful improvements in overall disease burden reported by both patients and physicians - ...