Biogen and Ionis Pharmaceuticals: A Message to the SMA Community

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 . . .

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Scarlett’s Night at MDA Muscle Team Gala

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 . . .

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FDA Will Review Nusinersen for Treatment of SMA

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 . . .

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Accessing Deflazacort: What You Need to Know

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 . . .

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Simply Stated: What is Muscular Dystrophy?

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 . . .

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Born Determined: 10-Year-Old Michigan Girl With Myotonic Dystrophy Defies Limits Every Day

myotonic dystrophy 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.

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