Researchers found that giving Duchenne muscular dystrophy (DMD) patients glucocorticoid therapy keeps them mobile for a longer period of time and reduces their risk of dying. Their study, “Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study,”…
Clay Matthews of the Green Bay Packers took to the field in Week 13 of the NFL season wearing a cause close to his heart on his feet, as he joined the football league’s “My Cause My Cleats” campaign to raise funds for Duchenne muscular dystrophy (DMD). “My…
Karen Haberberg, a New York portrait photographer, has taken tens of thousands of pictures throughout her career of families, couples, pregnant women, teenagers, and fashion models — not to mention dramatic landscape shots from Iceland to Indonesia. She’s also done several personal photo projects including “Benjamin,” a haunting portrayal…
Solid Biosciences announced the pending start of a first clinical trial to test the company’s leading investigational therapy – called SGT-001, a microdystrophin gene transfer candidate, as a potential treatment for Duchenne muscular dystrophy (DMD). The Phase 1/2 trial, called IGNITE DMD, will soon open for patient enrollment in the…
Capricor Therapeutics has received a green light from the U.S. Food and Drug Administration to launch a Phase 2 study of CAP-1002 cell therapy in children and adults with advanced Duchenne muscular dystrophy. Capricor expects to open the trial to patient enrollment in early 2018 and, if successful,…
The first healthy subject was dosed in a Phase 1b clinical trial evaluating the safety, tolerability and drug properties of ELX-02 as a candidate for the treatment of multiple conditions, including Duchenne muscular dystrophy (DMD). The multiple ascending dose (MAD) study (NCT03309605) is being led by Sevion…
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…
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