Two magnetic resonance measures — MRI and magnetic resonance spectroscopy (MRS) — of leg muscles are quality biomarkers that help…
Articles by Ana Pena, PhD
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to losmapimod, an investigational oral…
The investigational oral treatment losmapimod is able to suppress the toxic activity of the DUX4 gene, the root…
Parent Project Muscular Dystrophy (PPMD) has awarded two grants, one to further development of a gene therapy to prevent…
Catabasis Pharmaceuticals and the Jain Foundation have started a preclinical research collaboration to study edasalonexent…
Most parents of children with Duchenne muscular dystrophy and adults with this disease — who took part in an interview…
An adverse event report was erroneously submitted for a patient enrolled in Study-102 testing Sarepta Therapeutics’ micro-dystrophin gene…
The Muscular Dystrophy Association (MDA) awarded $389,000 in funding to a researcher from University of California,…
Early trial data supports the potential of FibroGen‘s investigative treatment pamrevlumab to slow the decline in, or…
Enrollment is progressing quickly in Catabasis Pharmaceuticals‘ pivotal Phase 3 PolarisDMD trial, testing its investigational small molecule…