Little Jamie Tierney Jr's parents have been desperately trying to save him since he was diagnosed in March 2022.

One of the demands raised during the protests was access to 'Elevidys', a one-time gene therapy treatment that has the ...

Managed Healthcare Executive gives C-suite executives in health plans and provider organizations news and strategies for ...

Sarepta Therapeutics (NASDAQ:SRPT – Get Free Report) was downgraded by stock analysts at Royal Bank of Canada from an ...

Matthew B. Klein, M.D., the Chief Executive Officer of PTC Therapeutics, expressed disappointment at the EC’s decision but ...

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

Keros plans on engaging with regulatory authorities, starting in the third quarter of 2025. Subject to the outcome of these regulatory interactions, Keros expects to initiate a Phase 2 clinical trial ...

WARREN, NJ, USA I March 28, 2025 I PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has adopted the opinion of the ...

PTC Therapeutics said on Friday the European Commission will remove its inherited progressive muscle-wasting disorder from the region after an advisory panel reaffirmed a negative opinion of the drug ...

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 ...

Cambridge, USA-based RNS specialist Wave Life Sciences (Nasdaq: WVE) has announced positive data from the Phase II FORWARD-53 trial of WVE-N531, which is an exon skipping oligonucleotide being ...