An expression of the live unlimited spirit, stories and voices of the MDA community
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 . . .
Aaron, 16, is an avid fan of rock music from ’70s to the ’90s — he looks up all the facts about songs, composers and artists as he is listening to them. “He is a veritable fount of trivia on rock music,” says his dad, Richard. Aaron, who was diagnosed with muscular dystrophy in early . . .
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 . . .
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 . . .
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 . . .
For individuals and families living with neuromuscular diseases, having access to the right support is paramount to living life to the fullest. Groundbreaking research, clinical trials and care from the best physicians are critical. But so is everyday help, and that’s where MDA Care Center social workers come in. Social workers can help individuals address . . .
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 . . .
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 . . .
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