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Thanks in large part to the work of MDA advocates across the country, the US House unanimously moved yesterday to pass the Newborn Screening Saves Lives Reauthorization Act (HR 2507).…

MDA has awarded MDA Venture Philanthropy (MVP) funding totaling $389,463 over two years to Rachelle H. Crosbie, PhD, professor and chair of Integrative Biology and Physiology at the University of…

Last week, the US House Committee on Energy and Commerce’s Subcommittee on Health considered the Newborn Screening Saves Lives Reauthorization Act (HR 2507), a bill that would reauthorize the national…

A little knowledge and preparation can get your travel plans off the ground

In recent years, drug developers have been able to pair the ever-expanding pool of gene identifications with advances in gene-targeted therapies. Researchers look to treat genetic disease at its source.

How these promising therapies are being studied and tested for neuromuscular diseases

A hiker proves she is stronger than FSHD by taking on the toughest trek of her life

Updates on some of the most promising studies

A Q&A with neurologist Tetsuo Ashizawa, M.D.

FDA Panel (left to right): Janet Woodcock, MD, FDA Center for Drug Evaluation and Research, and Peter Marks, MD, PhD, FDA Center for Biologics Evaluation and Research. FDA directors and…