Scientists at the Hospital for Sick Children (SickKids) and the University of Las Vegas Nevada (UNLV) have uncovered a genetic link between autism spectrum disorder (ASD) and a rare genetic condition ...
– 53.7% mean splicing correction observed following a single 15 mg/kg dose of PGN-EDODM1, with all patients showing an improvement in splicing – – PGN-EDODM1 was generally well-tolerated at 15 mg/kg, ...
BOSTON--(BUSINESS WIRE)--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of ...
Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people. While it is well known for causing muscle weakness and stiffness, DM1 also ...
- Primary endpoint is the five times sit to stand (5xSTS) test; secondary and exploratory endpoints will assess muscle function, CNS manifestations, and patient- and clinician-reported outcomes - - ...
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