Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.

Trial achieved key objectives for safety, tolerability, pharmacokinetics and pharmacodynamicsClinical data from this trial, together with ...

PTC Therapeutics said on Friday its inherited progressive muscle-wasting disorder drug will no longer be available for sale in the EU as the European Commission has decided not to renew its ...

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines ...

WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients ...

Wave Life Sciences Ltd. shares promising updates on AATD & DMD treatments. Click here for my updated look at WVE stock ...

Wave Life Sciences’ Phase II open-label trial investigating its disease-modifying drug for boys living with Duchenne Muscular ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death ...

Precision Biosciences Inc. recently presented a new gene-editing approach, PBGENE-DMD, which could allow life-long benefits to patients with Duchenne muscular dystrophy (DMD).

The Cambridge-based biotech's experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, ...

Amid readouts from the Muscular Dystrophy Association’s annual meeting, the Clinical Trials Arena evaluates five Duchenne trials in 2025.

The company plans to file next year for an accelerated clearance of its "exon-skipping" treatment, which would compete with one of Sarepta's medicines.