Despite the obstacles, Riley Herrera had a life filled with family, sports, travel, faith and hope before his death on Nov.

Entrada plans to initiate clinical testing of ENTR-601-44 in Duchenne MD patients amenable to exon 44 skipping later this year.

Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX) shares began Tuesday sharply higher. Cumberland, a specialty pharmaceutical company with development efforts focused on new products for rare diseases, ...

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from ...

Entrada Therapeutics has secured authorisation from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) to ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

BOSTON - Entrada Therapeutics , Inc. (NASDAQ:TRDA) received authorization from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial for its ...

Contrary to prior data, this study shows that neither eosinophils nor STAT6-mediated IL-4/IL-13 signaling contribute ...

Personalized organoids — ‘mini-organs’ — may accelerate treatment for patients with rare genetic disorders. Courtesy of Europeana via Unsplash Across the globe, countless babies ...

BOSTON - PepGen Inc. (NASDAQ: PEPG), a biotechnology firm focused on oligonucleotide therapies for severe neuromuscular and neurological diseases, is progressing with its CONNECT clinical program for ...

Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...

The positive results from the second part of the EMBARK trial contrast with findings from one-year post treatment, which did ...