Benitec Biopharma has submitted an application for orphan drug designation with the U.S. Food and Drug Administration (FDA) for its lead gene therapy candidate, BB-301, for the treatment of oculopharyngeal muscular dystrophy (OPMD). If granted, the orphan drug status will guarantee seven-year market exclusivity upon treatment approval, but…
Duchenne UK has teamed up with five pharmaceutical companies to start Project HERCULES, a collaboration aimed at persuading the national health insurance system to cover new Duchenne muscular dystrophy therapies. Ensuring that patients obtain access to innovative therapies is a two-part challenge. The first is obtaining regulatory approval of the drugs.
The absence of a signaling protein leads to immature muscle cells being unable to fuse together to promote muscle tissue growth, a study shows. University of Louisville researchers said lack of the MyD88 signaling protein prevented immature muscle cells known as myoblasts from fusing. When the protein’s levels are normal,…
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…
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