An expression of the live unlimited spirit, stories and voices of the MDA community
Earlier this month, in my home state of Colorado, I had the honor of attending the Denver MDA Muscle Walk with some amazing families and individuals. Not only was it great to be back home, it was wonderful meeting so many inspiring groups of people. It never ceases to amaze me how close everyone in . . .
Results from a natural history study for familial (inherited) ALS caused by a mutation in the SOD1 gene have confirmed that a variation of the mutation called SOD1 A4V is associated with a more aggressive disease course when compared to non A4V SOD1 ALS. A variety of mutations in the gene for the SOD1 protein account . . .
Osaka-based Mitsubishi Tanabe Pharma reported on June 20 that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration, seeking marketing approval of its drug edaravone to treat people with ALS (amyotrophic lateral sclerosis). The Japanese pharmaceutical company’s NDA submission marks one of its first steps in establishing a presence in . . .
When Dr. Ryan Wuebbles stepped into a lab in 2002 to study muscular dystrophy, he wasn’t just a bright-eyed, eager graduate student. He was also a young man who wanted to understand his own disease.
Peter Jones, associate professor in cell and developmental biology and neurology at University of Massachusetts Medical School in Worcester, was awarded an MDA research grant totaling $300,000 over a period of three years to develop a research mouse model for facioscapulohumeral muscular dystrophy (FSHD). Please describe your current research. Facioscapulohumeral muscular dystrophy (FSHD) is one . . .
Earlier this Spring, I had the honor of taking part in the 3rd annual Strength, Science and Stories of Inspiration event at the Harvard Science Center in Cambridge, Massachusetts. As a patient with dysferlinopathy (a form of muscular dystrophy), I was heartened to see every seat in the 500-person auditorium filled to support the mission of finding . . .
Editor’s note: The following is adapted from a letter Stephanie Hoover wrote to family and friends to ask them to support her in the Louisville Muscle Walk. We thought her characterization of the impact of her disease and her eloquent description of the community she has found within MDA were worth sharing. We hope you agree. . . .
Data released by the US Department of Labor this month showed that the unemployment rate for people with disabilities is more than double the unemployment rate of those who are considered able bodied. Many factors impact access to employment for those living with disabilities, including a system of benefits and services that are gained and . . .
Marathon Pharmaceuticals announced today it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the investigational drug deflazacort for the treatment of people with Duchenne muscular dystrophy (DMD). The FDA has 60 days to determine whether the NDA is complete and acceptable for filing. Deflazacort, a glucocorticoid, works . . .
Cohl Hometown: Exeter, NH Age: 9 Diagnosis: I was diagnosed with Spinal Muscular Atrophy, Type 3, at age 8. Spinal Muscular Atrophy (SMA) is a genetic disease affecting the part of the nervous system that controls voluntary muscle movement. Favorite School Subject/Activities: My favorite subject in school is social studies. Favorite People: My mom, dad, sister, Ava, and cat, Celine . . .
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