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 ...

"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 ...

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

The analysis indicates that in 2023, the US had approximately 43,255 diagnosed cases of Myotonic Dystrophy Type 1 and 10,815 cases of Myotonic Dystrophy Type 2. This data highlights a significant ...

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 ...

and notes that the news is reminiscent of what happened to PepGen when they initiated U.S. sites in the Phase 1 FREEDOM-DM1 study of PGN-EDODM1 in myotonic dystrophy type 1. Leerink is optimistic ...

Patients with myotonic dystrophy have smaller hearts, and lower systolic and diastolic blood pressures and pulse rates. They have impaired autonomic reflexes, measured by orthostatic, Valsalva ...

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

The analysis indicates that in 2023, the US had approximately 43,255 diagnosed cases of Myotonic Dystrophy Type 1 and 10,815 cases of Myotonic Dystrophy Type 2. This data highlights a significant ...