April 2026

Upsher-Smith Laboratories has launched two new websites to support people with Duchenne muscular dystrophy (DMD) who are taking its recently introduced corticosteroid therapy Kymbee (deflazacort). One website is designed specifically for caregivers of children with DMD. It offers tips for talking with healthcare providers about treatment, along with a step-by-step guide on how to start…

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Researchers at Shionogi Inc. are conducting a phase 2 clinical trial for adults with late-onset Pompe disease (LOPD) who are receiving standard-of-care enzyme replacement therapy (ERT). The study will evaluate the safety, effectiveness, and processing in the body of the investigational drug S-606001 when used as an add-on to ERT. S-606001 blocks an enzyme involved…

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Last week was a busy one for me. In addition to my normal activities, I took field trips on Tuesday and Thursday, looking for solutions to some of my recent struggles related to facioscapulohumeral muscular dystrophy (FSHD) and other health issues. I’m really struggling to get around our home using my walker. I get partway…

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A new collaboration aims to improve the design of clinical trials testing potential treatments for facioscapulohumeral muscular dystrophy (FSHD). The FSHD Society, a patient-driven advocacy organization, is teaming with two other groups — Solve FSHD, a venture philanthropic organization focused on speeding the development of new FSHD treatments, and the FSHD Clinical Trial Research Network, …

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“Don’t hope for a miracle. Make one,” is the powerful tag line from “Extraordinary Measures,” a 2010 dramatic film starring Brendan Fraser, Harrison Ford, and Keri Russell. John Crowley delivered the keynote address at the 2026 MDA Clinical & Scientific Conference. The movie is inspired by the true story of John and Aileen Crowley, a…

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Kathryn Arvidson champions accessible mental health treatment for people with disabilities.  Kathryn was diagnosed as a child with a rare degenerative neuromuscular disorder, Pompe disease, that impacts her mobility and pulmonary functioning.  Kathryn earned a master’s degree in social work and served as a Licensed Clinical Social Worker and supervisor for licensed professional counselors in…

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I have always been an open book about my life. I am a writer, caregiver, and mother living in the Midwest with my family. My husband and I have seven children: Lexi, 24; Max, 20; Chance, 18; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max, Rowen, and Charlie all live with Duchenne muscular…

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The U.S. Food and Drug Administration (FDA) has given Mesoblast the go-ahead to launch a clinical trial testing its cell therapy remestemcel-L-rknd in children with Duchenne muscular dystrophy (DMD). The trial aims to enroll 76 kids with DMD ages 5-9 who are receiving standard-of-care therapies. Participants will be randomly assigned to receive infusions of the…

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In this Quest Podcast episode, we chat with Emmy Award-winning filmmaker and disability advocate Samuel Habib and his father and longtime collaborator Dan Habib, the creative duo behind the extraordinary documentary The Ride Ahead. In the film, Samuel opens up about his personal journey into adulthood — navigating housing, employment, relationships, and higher education while…

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Quest Media is an innovative, adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities. With so many valuable…

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