A Pfizer result is unsettling the gene therapy push for Duchenne muscular dystrophy: The boys didn't improve even though a ...

While success is far from guaranteed in bio-land, with this week’s raising, Percheron has the horsepower to have a decent ...

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has once again snubbed Translarna ...

A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular ...

A study of 555 patients further solidified genotype-phenotype correlations in Duchenne muscular dystrophy (DMD).

On Friday, Capricor Therapeutics (NASDAQ:CAPR) announced 3-year safety and efficacy results from its ongoing HOPE-2 ...

Data Supports Deramiocel’s Sustained Efficacy and Safety in Treating DMD- -Improvements Seen in Multiple Cardiac and Skeletal ...

Capricor Therapeutics started its rolling submission seeking FDA approval of its DMD therapy deramiocel for heart muscle ...

Doha, Qatar: Sidra Medicine's Genetic and Genomic medicine clinic is providing life-altering gene therapy for rare genetic ...

EMA re-confirms non-renewal of conditional marketing authorisation of Duchenne muscular dystrophy medicine, Translarna: Amsterdam, The Netherlands Monday, October 21, 2024, 10:00 ...

Since its inception in 1992, the FDA’s accelerated approval pathway has helped shepherd nearly 300 new drugs to the market.