In recent years, a growing understanding of myotonic dystrophy type 1 (DM1)—an often fatal, devastating, rare neuromuscular disorder that causes muscle weakness and other life-limiting ...

People with Myotonic Dystrophy Type 1 (DM1), the most common adult-onset form of muscular dystrophy, progressively lose ...

global Phase I/II ACHIEVE trial in adults with myotonic dystrophy type 1 (DM1). The ACHIEVE trial currently includes 56 participants and is fully enrolled through the 6.8 mg/kg Q8W cohort ...

EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD), and is still ongoing in the United Kingdom.

Arrakis Therapeutics Inc. has presented data on its RNA-targeted small-molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1). The company’s proprietary RNA‐specific chemical ...

The FDA has put a stop to U.S. initiation of PepGen’s Phase II trial for its Duchene muscular dystrophy treatment. The ...

"Dune"-inspired Arrakis Therapeutics has tested its spice—an RNA-targeting small molecule—in a mouse model of myotonic dystrophy type 1 (DM1), with the drug reversing the prolonged muscle ...

Type-Specific Diagnosed Prevalent Cases of Myotonic Dystrophy 1, Age-Specific Diagnosed Prevalent Cases of Myotonic Dystrophy, and Comorbidity Associated With Diagnosed Prevalent Cases of Myotonic ...

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Dr. Reshma Kewalramani, Chief Executive Officer and President, will present at the 43rd Annual J.P. Morgan Healthcare Conference ...

It develops a broad portfolio of therapeutics for muscle diseases, including lead programs in myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral muscular dystroph.