Strongly


An expression of the live unlimited spirit, stories and voices of the MDA community

Ethan Lybrand, MDA Summer Camp, and His Wheelchair Named “Bumblebee”

Ethan Lybrand loves his Bumblebee. On Oct. 27, the 7-year-old became the proud owner of his first power wheelchair. Black and yellow, the chair’s color scheme is inspired by, and named after, his favorite Transformers character, Bumblebee. For Ethan, who has Duchenne muscular dystrophy, getting a wheelchair could have been a tough transition. Instead, Ethan . . .

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Participants Sought for Clinical Trial to Test Vamorolone in DMD

Researchers are seeking volunteers to participate in a clinical trial sponsored by ReveraGen BioPharma to test the safety and effectiveness of the investigational drug vamorolone in boys with Duchenne muscular dystrophy (DMD). In the two-part, open-label study called “A Phase 2a and 2a Extension study to Assess Vamorolone in Boys with Duchenne Muscular Dystrophy,” all . . .

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Encouraging Results in Nusinersen (Spinraza) Trial for Type 2 SMA

In a joint statement today, Biogen and Ionis Pharmaceuticals announced encouraging interim 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 . . .

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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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Why Multidisciplinary Care Matters

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 . . .

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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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