When Zac Henderson could no longer play baseball, he joined the school jazz band. When he could no longer hold his instrument, he migrated to the local 4-H. Then it was on to managing his high school’s basketball team. Now Zac has his own film production company thanks to working on the basketball team’s highlight . . .
Today we couldn’t be happier as, together with the Duchenne community, we celebrate FDA accelerated approval of eteplirsen to treat kids and adults with some forms of Duchenne muscular dystrophy (DMD). “This is something that we’ve watched, and hoped and prayed for, and seen it develop over the last seven years or so,” said Josh . . .
Over the last six decades, MDA has committed $1 billion to research designed to move the world closer to treatments and cures for muscular dystrophy, ALS and related life-threatening diseases. As a result, MDA’s fingerprints are on nearly every major neuromuscular disease research breakthrough, and the progress we continue to see in the field is . . .
Rachelle Crosbie-Watson, professor and vice-chair of graduate education at the University of California Los Angeles, was awarded an MDA research grant this summer totaling $300,000 over a period of three years to test new therapies for cardiomyopathy (heart muscle weakness) associated with Duchenne muscular dystrophy (DMD). Can you tell us a little bit about your . . .
Noah Hometown: Cortland, NY Age: 17 Diagnosis: I was diagnosed with Duchenne Muscular Dystrophy (DMD) Favorite School Subjects/Activities: My favorite school subject is history. Favorite People: My favorite people are my parents and my sister Katie. I also love spending time with my grandpa and my cat, Smokey, who is 16 years old. Interests: I like to read and I also . . .
Since its inception, MDA has invested more than $1 billion in neuromuscular disease research to uncover new treatments and cures. In 2016 alone, MDA awarded 66 new research, development and research infrastructure grants with a total funding commitment of more than $17 million. These and other MDA grants fund research projects designed to uncover the . . .
This summer MDA challenged our families, friends, sponsors, staff and others around the nation to share their stories about living life without limits despite the challenges of neuromuscular disease. Some shared moments tied to pursuing an education or career that was thought to be out of reach. Some talked about skydiving, hiking or running a . . .
Marathon Pharmaceuticals has announced pivotal Phase 3 data evaluating the investigational drug deflazacort for the treatment of Duchenne muscular dystrophy in the journal Neurology. The study results show that deflazacort demonstrated significant improvement in muscle strength compared with placebo in 12 weeks. In addition, results show that deflazacort was associated with less weight gain than . . .
Researchers are looking for boys and men living with Duchenne muscular dystrophy, ages 12 years and older, to participate in a phase 2 clinical trial, sponsored by FibroGen, to test the experimental drug FG-3019 (pamrevlumab). The study, known as “MissionDMD,” is designed to help researchers evaluate whether FG-3019 is safe and effective in people with . . .
Starting a new school year is hard on every parent and child, but it’s particularly difficult when you have a child with special needs. As a parent you want to make sure that those needs are met all day every day, even if you are not with them. Even after you explain what your . . .
