News

Research into molecules that appear to target the gene known to cause facioscapulohumeral dystrophy (FSHD) may lead to a treatment able to slow disease progression, a study reports. The study, “BET bromodomain inhibitors and agonists of the beta-2 adrenergic receptor identified in screens for compounds that inhibit DUX4 expression…

Exonics Therapeutics has secured $40 million in financing from The Column Group (TCG) — a science-driven venture capital firm — to develop its lead gene editing program in Duchenne muscular dystrophy (DMD). Exonics, based in Cambridge, Massachusetts, is working to develop a safe, one-time gene editing treatment that would provide lifelong…

In recent years scientists have learned that the protein utrophin can substitute for the dystrophin protein that is missing in muscular dystrophy. Researchers have now discovered that utrophin can reduce damage to energy-producing cell components known as mitochondria, whose deterioration is linked to MD. The reduction in damage improved the muscle…

The U.S. Food and Drug Administration has taken a step that will allow Sarepta Therapeutics to start clinical trials of its Duchenne muscular dystrophy therapy SRP-5051. That move was accepting the company’s Investigational New Drug application for SRP-5051. The therapy, known as a peptide phosphorodiamidate morpholino oligomer, is designed to…

Wave Life Sciences has started a Phase 1 clinical trial of a treatment for a gene malfunction linked to the most common form of Duchenne muscular dystrophy. Genes consists of dozens of components called exons. If an exon is missing, a gene can fail to produce a protein the body needs.

The U.S. Food and Drug Administration (FDA) has cleared an investigational new drug application for a micro-dystrophin gene therapy program by Sarepta Therapeutics and Nationwide Children’s Hospital, in clinical development for Duchenne muscular dystrophy (DMD). A Phase 1/2a clinical trial is now enrolling participants and should begin dosing…

The Hope for Gus Foundation and its signature Everest to End Duchenne fundraising trek to Mount Everest Base Camp have raised $100,000 to fund new gene-editing research that could lead to new treatments for Duchenne muscular dystrophy (DMD). The nine-member U.S. Everest to End Duchenne team…

The U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for the GALGT2 gene therapy program for Duchenne muscular dystrophy (DMD), Sarepta Therapeutics and Nationwide Children’s Hospital recently announced. Following the FDA clearance, Nationwide Children’s will continue to advance the program by initiating a…

A new method using DNA isolated from a blood sample was far more sensitive in detecting large mutations causing Duchenne muscular dystrophy than currently used genetic diagnostics tools, according to a new study. The next-generation mapping method, using the Bionano Genomics platform, has the potential to replace less sensitive…

Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…