– 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, ...
- Preclinical data show ARO-DM1 reduces muscular DMPK expression and corrects spliceopathies, which could lead to improved muscle strength and function Patients with DM1 have muscle weakness and ...
– Lifting of FDA hold allows FREEDOM-DM1 study to launch in the U.S. with target dose levels of 5 mg/kg, 10 mg/kg and 20 mg/kg – – Safety, transcript splicing and clinical outcome measures data at 5 ...
Mechanisms of molecular pathology and erythromycin treatment in myotonic dystrophy type 1 In myotonic dystrophy type 1, aberrant RNA transcribed from the mutant DMPK gene leads to the aggregation of ...
Researchers focused on Myotonic Dystrophy 1 heart problems are testing a novel approach to restore normal function. Myotonic Dystrophy Type 1 (DM1) is the most common adult-onset form of muscular ...
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 ...
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