Madhuri Hegde, associate professor in the department of human genetics at Emory University in Atlanta, was awarded an MDA research infrastructure grant totaling $300,000 over a period of three years to continue groundbreaking work to identify and characterize new gene defects that can cause limb-girdle muscular dystrophy (LGMD). In the era of precision medicine, it . . .
In the fall of 2009, I met a girl. Not in the traditional way — I met Denise via eHarmony. Yes, we were an eHarmony success story. We often joked about the eHarmony commercials with the two incredibly good-looking people dancing in a beautiful garden or around a fountain. We thought how cool it would . . .
If there was one piece of advice MDA Resource Specialist Ashleigh Peska could offer individuals with muscular dystrophy and other muscle-debilitating diseases, it would simply be, “don’t quit!” Ashleigh, who lives with limb-girdle muscular dystrophy (LGMD), is a shining example of this mantra. A college graduate and former MDA Iowa State Goodwill Ambassador, Ashleigh has . . .
On Sept. 19, the U.S. Food and Drug Administration granted accelerated approval to eteplirsen, which will now be marketed under the brand name Exondys 51 for the treatment of some forms of Duchenne muscular dystrophy (DMD). We’ve received a lot of questions about the newly approved drug, and wanted to share a few answers to . . .
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 . . .
MDA and Target ALS Foundation have announced a partnership aimed at advancing ALS research and therapy development. Through the partnership, the two organizations will work together to support talented young scientists who choose to pursue a career devoted to ALS research. The partnership represents a key step in MDA’s commitment to form strategic alliances with . . .
Meet Sylvia Hometown: Oakland, CA Age: 15 Diagnosis: I was diagnosed with Bethlem myopathy. Bethlem myopathy is a type of limb-girdle muscular dystrophy—a group of disorders affecting voluntary muscles, mainly those around the hips and shoulders. Favorite School Subject/Activities: My favorite subjects are leadership and geometry. Favorite People: My favorite person is Ellen DeGeneres and I also love my dogs Ronnie and . . .
In a joint statement today, Biogen and Ionis Pharmaceuticals announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Submission of the NDA is the first step toward getting approval . . .
MDA Lock-Up Jailbirds can earn their chance to win a pair of roundtrip airline tickets, Beats wireless headphones or an iPad Air 2. For every $100 Jailbirds raise between now and Friday, November 18 they will be automatically entered into the drawing.* Jailbirds are encouraged to track their progress at their Participant Center, and check . . .
This September as we recognize Newborn Screening Awareness month, we are proud to announce a new partnership with Cure SMA aimed at moving newborn screening forward for spinal muscular atrophy (SMA)—the leading genetic cause of death in infants. This is an exciting time for the neuromuscular disease community as decades of research are translating into . . .
