News

LAMA2 Muscular Dystrophy Drug Candidate TXA127 Is Granted Orphan Drug Status

Tarix Orphan LLC recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to TXA127, the company’s lead candidate for the treatment of laminin-deficient congenital muscular dystrophy (LAMA2 MD). Early-onset LAMA2-related muscular dystrophy (MD) occurs in nearly one in every 30,000 people and is responsible for…

Muscle Proteins May Serve as Serum Biomarkers for 3 Types of Muscular Dystrophy

Pfizer, in collaboration with Newcastle University, reports finding four serum biomarkers for three types of muscular dystrophy. The markers may be valuable for monitoring disease progression and treatment response in both preclinical and clinical studies. Researchers analyzed previously collected serum samples from three types of muscular dystrophies — 38 Becker muscular dystrophy (BMD) and…

Duchenne MD Therapy by Gene Editing of Stem Cells May Be Possible in Next Decade

UCLA scientists have developed a potential gene therapy approach for Duchenne muscular dystrophy (DMD) using CRISPR/Cas9 technology and stem cells. If the new treatment proves successful and reaches the clinic — possibly in the next decade — it could be applied to 60 percent of all Duchenne patients. CRISPR/Cas9 is a gene-editing technology that allows targeting and…

FDA Extends Its Review of Potential DMD Therapy, Eteplirsen, into May

Sarepta Therapeutics, a developer of RNA-targeted treatments, announced that the U.S. Food and Drug Administration (FDA) is extending by another three months its review of the company’s New Drug Application (NDA) for eteplirsen, a potential treatment for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. A reason for the extension was…