A Phase 2 trial of ATL1102 met its primary goal of demonstrating favorable safety and tolerability in boys with Duchenne…
Articles by Joana Carvalho, PhD
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Epirium Bio’s EB 002 ((+)-epicatechin) for…
Capricor Therapeutics will host a conference call and webcast with a cardiac expert to discuss heart complications associated with…
Fulcrum Therapeutics announced that its Phase 2b trial assessing the safety and efficacy of losmapimod, an investigational oral…
Treatment with chloroquine increased the levels and restored the activity of Muscleblind-like RNA-binding proteins, improving muscle function and…
The gene-editing tool CRISPR/Cas9 was used to correct defects in the DMD gene and restore dystrophin protein production, lengthening the…
GeneDx has chosen Bionano Genomics’ technology, called Saphyr System, to aid in developing tests to detect mutations…
Note: This story was updated Dec. 17, 2020, to note that preclinical studies testing this form of gene therapy were…
Solid Biosciences announced it will reduce its workforce by approximately one third to cut expenses and focus on…
Astellas Pharma recently agreed to acquire Audentes Therapeutics, a move it expects will result in faster development of potentially…