Jon Anning, a Slippery Rock University professor, has done extensive research on effects and treatment for Duchenne muscular ...

Orphan drug designation from the European Commission underscores Solid’s commitment to advance SGT-003 through a global ...

Santhera Pharmaceuticals (SIX: SANN) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending ...

Santhera Pharmaceuticals (SIX:SANN, OTC:SPHDF, FRA:S3F0), the Swiss rare disease specialist, has received a positive opinion ...

The Phase 1/2 FUNCTION-DMD study will enroll ambulatory patients and is designed to evaluate safety, tolerability, and efficacy, including dystrophin protein expression and functional outcomes. "The ...

Duchenne muscular dystrophy (DMD) is a genetic condition caused by a change, or mutation, in the DMD gene. This gene normally tells the body how to make dystrophin, a protein that helps protect ...

Gene therapies for DMD offer potential benefits but are not curative and carry risks, necessitating informed patient and caregiver perspectives. Early diagnosis through newborn screening raises ...

DURHAM, N.C.--(BUSINESS WIRE)-- Precision BioSciences, Inc. (DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for ...

Solid Biosciences announced today that new data from two preclinical studies reinforce the potential of its investigational microdystrophin gene therapy, SGT-001, to be an effective treatment approach ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the deterioration of skeletal muscle in the disease. The use of ...

STATEN ISLAND, N.Y. — Those who participated in “The 13th annual Michael 2025 Step For A Cure 5k Run And Walk” at the FDR Boardwalk and promenade were there to make a difference in the fight against ...