Participants Sought for FIREFISH SMA Study
Researchers are looking for individuals with type 1 SMA for a clinical trial to test the investigational drug RG7916.
Tag: Spinal Muscular Atrophy (SMA)
Participants Sought for SMA Gene Therapy Study
Researchers are looking for individuals with type 1 SMA to test efficacy for its SMA gene replacement therapy AVXS-101.
Magic Wheelchair Makes Mario Kart Halloween Magic for Boy with DMD
Iker, who lives with DMD, got his dream Halloween costume this year with a little bit help from Magic Wheelchair!
Sawsan Abuosbie, Who Lives with SMA, Talks College, Job Hunting and Work Life
In recognition of National Disability Employment Awareness Month, Sawsan, who lives w SMA, shares her experience of joining the work force.
Jerry Lewis’ Work Helped Scientists Discover the DMD Gene
In October 1986 Jerry Lewis announced the DMD gene had been discovered, a monumental moment in MD research.
With One Finger, AJ Brockman Paints the World
AJ Brockman, an artist who lives with SMA, uses only the index finger on his left hand to create works of art.
Debunking Myths about SMA
MDA National Ambassador Joe Akmakjian debunks a few myths about SMA in recognition of SMA Awareness Month.
Five Questions with Researcher Lyndsay Murray
Lyndsay Murray of the University of Edinburgh in Scotland was awarded an MDA research grant to investigate how therapies work at different stages of SMA
“A Lot of People Care About Me:” Muscle Walk Raises Money and Teen with Myotonic Muscular Dystrophy’s Spirits
After Erica Destache was diagnosed with myotonic muscular dystrophy, her family raised a whopping $20,000 for their local MDA Muscle Walk.
Access to Health Care and to Approved Therapies is Essential for MDA Families
Despite the progress in the quest for treatments and cures, individuals living with the life-threatening diseases that newly approved drugs treat are facing a new challenge — gaining access to the approved therapies. Today, some individuals and families are unable to access FDA approved therapies, which can significantly impact the health and life of the person denied access. Not having access to appropriate therapies is unacceptable. A treatment cannot change the lives of families and individuals if they do not have access to it.
