News

The researchers who developed the CRISPR DNA editing method that has taken medicine by storm have come up with a tweak to the technique that has implications for Duchenne muscular dystrophy. They can now edit single units of RNA code without modifying genetic content. RNA are building blocks of life that…

Summit Therapeutics announced the completion of a 24-week dosing period of ezutromid, its investigational therapy for patients with Duchenne muscular dystrophy (DMD). Summit expects to report the results of this initial dosing period of its Phase 2 trial during the first quarter of 2018. The Phase 2 PhaseOut DMD…

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…

A one-time infusion of Capricor Therapeutics’ heart progenitor cell therapy CAP-1002 improved heart muscle function and reduced tissue scarring in a small group of patients with Duchenne muscular dystrophy. The impact of the therapy, however, went beyond heart improvements, as the study demonstrated that CAP-1002 also improved hand and…

•The Muscular Dystrophy Association has awarded 13 grants totaling $3.5 million to researchers working on treatments for neuromuscular diseases such as MD and ALS, studies that identify the diseases’ causes, or research on the disorders’ underlying mechanisms. This brings the total amount of the grants the organization has made this…

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.