In a joint statement today, Biogen and Ionis Pharmaceuticals announced encouraging results from the late-stage CHERISH clinical trial to test the experimental drug nusinersen (brand name Spinraza) in children with type 2 spinal muscular atrophy (SMA). Based on the results, investigators have stopped the trial to allow all participants to transition into an open-label trial . . .
Hockey season is finally underway, and the Chicago Blackhawks have added one more fan to their roster. No, this fan is not a burly teenager who loves to play hockey, or someone mesmerized by the Blackhawks and their Stanley Cup glory of the past six years. This fan is 5-year-old Scarlett Sepe, an adorably outgoing . . .
Jake Roszelle is a “go big or go home” type of guy. Between Jake and his brother, their mom, Krista, is running out of room for all the plaques and medals. “He’s a very spirited go-getter,” Krista says of Jake. “He just never took his disability to heart, I guess. He works 100 percent of . . .
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 . . .
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 . . .
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.