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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 ...

Entrada Therapeutics has secured the Health Authorities and Ethics Committees' authorisation within the European Union (EU) ...

Each genetic sequence contains part of the final gene ... Meanwhile, Pfizer ditched its DMD gene therapy candidate in July after it, too, failed a phase 3 trial, which led to multiple rounds ...

PARIS--(BUSINESS WIRE)--Genethon unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne Muscular Dystrophy (GNT-016-MDYF) at the annual meeting of the ...

It is an X-linked recessive disorder that occurs due to changes in the DMD gene, which codes for the protein dystrophin. Dystrophin is a protein that plays a role in strengthening muscle fibers ...

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 ...

Gene therapies have set a high bar for outcomes in pharma by delivering curative treatments for difficult diseases in a one-and-done punch. But for Duchenne muscular dystrophy, the results of ...

RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin, a protein that is missing or defective in DMD patients. DMD is a form of ...

The FDA's recent expanded approval of delandistrogene moxeparvovec (Elevidys) widened access to the gene therapy to include ambulatory and non-ambulatory Duchenne muscular dystrophy patients ages ...