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Back in June 1984, a doctor calmly informed Pat Furlong that her sons — 4-year-old Chris and 6-year-old Patrick — had Duchenne muscular dystrophy. It was the first time she had ever heard those three words uttered together. The disease eventually claimed both boys, but that didn’t stop Furlong’s fight against…

The Muscular Dystrophy Association (MDA) awarded $389,000 in funding to a researcher from University of California, Los Angeles (UCLA) to support the development of a small molecule that holds promise for preventing muscle damage in Duchenne muscular dystrophy (DMD). The molecule raises levels of a…

A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…

CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD), significantly improved skeletal muscle and lung function in a group of boys and young men at advanced stages of the disease, top-line data from an interim analysis of a Phase 2 trial show. CAP-1002…

PF-06939926, Pfizer‘s experimental gene therapy for the treatment of patients with Duchenne muscular dystrophy (DMD), shows promising safety and efficacy results in a small group of boys with DMD, a Phase 1b trial shows. The company presented the preliminary findings at the 25th Annual Parent Project…

Treatment with Exondys 51 (eteplirsen) significantly slows respiratory decline in boys with Duchenne muscular dystrophy (DMD), including those with more advanced disease, compared to standard glucocorticoid therapy, an analysis of clinical trials shows. The study “Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy”…

For more than 20 years, Robert (Bob) Bennett was a steadfast leader and supporter of the Muscular Dystrophy Association (MDA). To honor his legacy, the Robert M. & Marjie A. Bennett Foundation has donated $500,000 to the Los Angeles-area MDA Summer Camp. Located in Orange County, Calif., the…

The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have scared off all but the most determined. At that time, few doctors in the developing country of 2 million — now called North Macedonia — had…

Early trial data supports the potential of FibroGen‘s investigative treatment pamrevlumab to slow the decline in, or even improve, lung and heart function in non-ambulatory youth with Duchenne muscular dystrophy (DMD), as well as to preserve their upper limb performance and strength. These findings were highlighted…

The Muscular Dystrophy Association (MDA) has awarded Locana $550,000 through its MDA Venture Philanthropy (MVP) program to help advance the biotech company’s therapy platform for myotonic dystrophy (DM). Specifically, the grant to the San Diego, California-based gene therapy company is aimed at furthering the development of its RNA-targeting…