- 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 ...
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 ...
– 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 ...
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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