The ALS Association, ALS Finding a Cure (ALSFAC), and MDA announced they have jointly awarded a clinical trial grant totaling more than $2.5 million over two-and-a-half years to leading investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital. The principal investigator is Stanley Appel, MD, co-director of Houston Methodist Neurological Institute, chair of . . .
Scholar Rock Holding Corporation announced positive results from its phase 1 clinical trial evaluating SRK-015, a therapy for treating spinal muscular atrophy (SMA), at the Cure SMA Annual Conference held June 28 through July 1 this year. Consistent with previously announced interim findings in February, the final results showed positive safety, pharmacodynamic, and pharmacokinetic data in healthy volunteers, supporting . . .
There are still spots available for MDA’s Public Policy and Advocacy Conference, taking place in Washington, DC, from Oct. 15-17 at the Grand Hyatt Washington. If you have been thinking about attending, now is the time to sign up before all the spots are filled! Click here to register. The MDA Public Policy and Advocacy . . .
The muscular dystrophies are a group of neuromuscular diseases that cause weakness and degeneration of the skeletal (voluntary) muscles. Muscular dystrophies are generally considered genetic diseases, or diseases that arise from genetic mutations, which can be either inherited or spontaneous. Some muscular dystrophies have been found to have a genetic component, which means a genetic . . .
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). Once enacted, this law will reauthorize the national newborn screening program for five years and make additional investment in order to modernize and make improvements . . .
We believe in the power of community and the importance of building relationships with families going through similar experiences. If you are an individual living with a neuromuscular disease, we invite you and your loved ones to attend one of our MDA Engage education events taking place across the country. MDA Engage Community Education Seminars . . .
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 California, Los Angeles. The award will support the development of a small molecule drug that increases expression of sarcospan, a protein that may help to . . .
On July 20, there will be burpees, pushups, lunges, and squats. Sweat will bloom on shirts, heartbeats will race, and grins will break out between breaths. This will be business as usual for members of the fitness franchise Burn Boot Camp, but there’s a twist — this time participants will be giving back while getting . . .
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 newborn screening program for five years. We are happy to report that the subcommittee unanimously agreed to send the bill to the full committee for . . .
July 9 at the Irvine Ranch Outdoor Education Center in Orange, Calif., home to one of MDA’s nationwide Summer Camp programs, was a very good day. Campers and MDA staffers gathered with members of the Bennett Foundation, who graciously donated $500,000 to the camp in honor of the late Robert (Bob) Bennett, president of MDA . . .