Clara was 2 years old when she was diagnosed with congenital muscular dystrophy. Her family was referred to their local Minneapolis MDA Care Center, and when they got there, Clara’s mom, Becky, says it felt “a bit like coming home.” Our MDA Care Center is a huge gift for us as working parents managing a . . .
The U.S. Food and Drug Administration (FDA) has accepted Biogen’s New Drug Application for nusinersen (brand name Spinraza) for the treatment of spinal muscular atrophy (SMA) and granted Priority Review. Priority Review status is reserved for drugs that offer significant improvements over existing options or provide a treatment for a disorder for which no approved . . .
When Jayden Long learned four years ago that the firefighters in his hometown of Nampa, Idaho, had to work on Christmas, he devised a plan. “He wanted to deliver baked goods to the firefighters on Christmas day,” recalled his mother, Shellie. “So we baked just about all night and delivered trays of food to three . . .
Every year at the end of October, millions of kids dress up in funny clothes to take over the streets in communities across America on a mission to collect all the candy. Sweets aside, for most kids, part of the fun is making an important decision: what they’ll be for the big candy collection. For . . .
The U.S. Food and Drug Administration (FDA) in August accepted Marathon Pharmaceuticals’ New Drug Application for deflazacort for the treatment of Duchenne muscular dystrophy (DMD) and granted Priority Review. A decision on deflazacort, which previously has received Fast Track status, Orphan Drug designation and Rare Pediatric Disease status from the FDA, is anticipated in February . . .
Mark Eisenberg was diagnosed with Duchenne muscular dystrophy at the age of 4, but he hasn’t let it stop him from achieving his dreams. He recently graduated from college, where he became largely involved in muscle disease awareness. He now works as a blogger, raising awareness through his current blog, Duchenne Talk. Two years ago . . .
Muscular dystrophy is a term that refers to a number of diseases that cause progressive loss of muscle mass resulting in weakness and, sometimes, loss of mobility. There are many different kinds of muscular dystrophy, each affecting different groups of muscles. In some types of muscular dystrophy, symptoms begin in childhood. In other forms, symptom . . .
We would like to thank MDA’s Maine State Goodwill Ambassador Jared Conant for allowing us to share his powerful speech made at this year’s MDA Muscle Walk in Maine. “Our devotion to ending all muscle disease is making progress. Yet each day, we need to find the strength to continue to push forward with our . . .
MDA and RYR-1 Foundation have announced a partnership aimed at advancing research and clinical care, raising awareness and improving education of patients, medical professionals and the public about RYR1-related myopathies. The partnership represents a key step in MDA’s commitment to form collaborative relationships with other organizations working on the same diseases MDA covers. “MDA is . . .
Kayla living unlimited by running on the track
Fabiola and Matthew watched their daughter run one lap. Then two. Then three. Kayla rounded the same bend again and again, a look etched on her face that her parents knew well — determination.
All in all, the 10-year-old ran nine laps at the Grand Rapids, Mich., Juniors River Bank Run. Other kids may have run more, but to Kayla and her parents, those nine laps were a marathon. They were as big as beating Usain Bolt in the 100-meter.
