The FDA has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne ...

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...

Precision BioSciences, Inc. received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for PBGENE-DMD to treat Duchenne muscular dystrophy (DMD).

Investing.com -- Precision BioSciences, Inc. (NASDAQ: DTIL) stock surged 17% on Wednesday after the company announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric ...

In North Carolina, 2 years of newborn screening began with CK-MM, followed by total creatine kinase and next generation sequencing of an 86-neuromuscular gene panel, including the DMD gene, if ...

Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), ... Each genetic sequence contains part of the final gene attached to a ribozyme sequence.

But because the DMD gene is so large, drug developers created a tiny version called microdystrophin that can fit into an adeno-viral vector. In theory, this gene replacement would impact the disease.

Sarepta’s shares crashed 41% in premarket trading Monday morning to $21.01 after the biotech reported a second death from ...

How Is Duchenne Muscular Dystrophy Diagnosed? DMD is diagnosed through a physical evaluation and blood tests to identify ...

"Regenxbio’s DMD gene therapy drives 122.3% biomarker uptick in three-year-old" was originally created and published by Clinical Trials Arena, a GlobalData owned brand.

Duchenne muscular dystrophy (DMD) is the most common type. It’s caused by flaws in the gene that controls how the body keeps muscles healthy. The disease almost always affects boys, and symptoms ...