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 ...

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 ...

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 ...

Avidity Biosciences' AOC platform shows promising results for genetic diseases, making it a strong buy with potential upside ...

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 ...

"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 ...

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

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 ...

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 ...