PBGENE-DMD, a novel first-in-class gene editing therapy, utilizes a gene excision approach that is clearly differentiated from existing microdystrophin and exon skipping treatments. PBGENE-DMD is ...
NASHVILLE, Tenn., March 19, 2025 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on rare diseases, today announced ...
"As we prepare to commence the Phase1/2 FUNCTION-DMD clinical trial for PBGENE-DMD, we're excited to present new preclinical data highlighting its potential to deliver durable, long-term functional ...
While PepGen works to persuade the FDA to let it launch a phase 2 trial of its Duchenne muscular dystrophy (DMD) therapy in the U.S., the biotech has decided to pause the U.K. arm of the study. The ...
A multidisciplinary team has proposed respiratory care guidance for pediatric and adult patients living with Duchenne muscular dystrophy (DMD) in the United Kingdom (UK), with these recommendations ...
DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing ...
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