An expression of the live unlimited spirit, stories and voices of the MDA community
Allison Willis and her sons, Nolan and Jack (15), who participated in the clinical trials for the exon-skipping drug Exondys 51 (eteplirsen), react to the FDA’s approval and expanded use of the drug. The drug treats Duchenne muscular dystrophy. Watch the video of their story and the groundbreaking clinical trial that made history:
A couple of years ago MDA got to know the Somers family… While Crystal and Cooper continue to mourn the loss of an incredible husband and father, they are able to focus on and appreciate the positives in their life now. Cooper recently turned 6, started first grade and is ready – and excited – . . .
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 . . .
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