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Exon-skipping therapy shows promise for DMD44
Delpacibart zotadirsen (del-zota), an investigational exon-skipping therapy from Avidity Biosciences, is demonstrating the potential to reverse disease progression in boys and young men with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping, or DMD44. That’s according to one-year data from the Phase 1/2 EXPLORE44 trial (NCT05670730) and the EXPLORE44 open-label extension (OLE) study (NCT06244082),…
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Exon-skipping therapy shows promise for DMD44
Delpacibart zotadirsen (del-zota), an investigational exon-skipping therapy from Avidity Biosciences, is demonstrating the potential to reverse disease progression in boys and young men with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping, or DMD44. That’s according to one-year data from the Phase 1/2 EXPLORE44 trial (NCT05670730) and the EXPLORE44 open-label extension (OLE) study (NCT06244082),…
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The how and why of living with limb-girdle muscular dystrophy
“He who has a why to live can bear almost any how.” — Friedrich Nietzsche I love a good quote as much as the next person. When I was teaching music in middle school, I’d sometimes write inspirational or thought-provoking quotes on the whiteboard to encourage conversation or reflection. At times, I’d have the kids find…
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Employer Matching Programs Double the Impact
Anybody who has made a monetary donation to support MDA’s mission knows well that their dollars go a long way to change the lives of those living with neuromuscular disease. But what many might not know is that corporate matching programs create an opportunity to double that donation – and double the impact. Companies with…
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Agamree : un premier traitement approuvé au Canada pour la DMD
Le 25 septembre 2025, Santé Canada a franchi une étape historique en approuvant AGAMREE® (vamorolone) comme traitement pour les garçons atteints de la dystrophie musculaire de Duchenne (DMD) âgés de 4 ans et plus. Jusqu’à présent, aucun médicament spécifique à la DMD n’était officiellement approuvé au Canada. Les familles devaient compter sur l’usage de corticostéroïdes,…
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Just another Monday: Watch movie, do laundry, throw out my back
Mondays are one of my favorite days of the week as a busy mom to seven children: Lexi, 24; Max, 19; Chance, 18; Rowen, 16; Charlie, 14; Mary, 11; and Callie, 3. I’m also a caregiver to Max, Rowen and Charlie, who all live with Duchenne muscular dystrophy (DMD). Because the weekends are jam-packed with…
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CureDuchenne Cares Webinar: Optimizing Cardiac Outcomes in Duchenne
CureDuchenne and Andreas S. Barth, MD, PhD, FAHA, for a community webinar about Optimizing Cardiac Outcomes in Duchenne. Watch HERE The post CureDuchenne Cares Webinar: Optimizing Cardiac Outcomes in Duchenne appeared first on CureDuchenne.
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Motor function linked to weight, height changes in boys with DMD
For boys who start corticosteroids to treat Duchenne muscular dystrophy (DMD), being taller is linked to slower growth, while being older is linked to more weight gain as they move into adolescence and near the loss of their ability to walk, according to Phase 3 clinical data. This implies that doctors should consider a boy’s…
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MDA Ambassador Guest Blog: Entrepreneurship, Advocacy, and Innovation: Building a More Accessible Future from the Inside Out
Owen Kent is the co-founder and Chief Marketing Officer of Assistive Technology Development (ATDev), a startup creating innovative mobility devices that empower people to live independently and confidently. As a lifelong wheelchair user with spinal muscular atrophy (SMA) Type II, Owen draws on his lived experience to design user-driven technologies, advocate for disability rights, and…
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MDA Ambassador Guest Blog: Helpful Tips for Accessible Halloween Costumes
Megan Jennings, known professionally as Sybil Thorn, is an artist and freelance creator. Born with spinal muscular atrophy, she received her bachelor’s degree in Theater and English with an emphasis in Creative Writing from Presbyterian College, where one of her favorite classes was in costume design. As a lifelong costumer, she looks forward to Halloween…
