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Avidity’s del-desiran for DM1 named orphan drug in Japan
The Japan Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug status to delpacibart etedesiran, known as del-desiran, an investigational therapy from Avidity Biosciences now in clinical testing for myotonic dystrophy type 1 (DM1). This designation is intended to support the development of therapies for rare diseases affecting fewer than 50,000 patients in…
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Travelers with Disabilities Share Their Top Tips to Avoid Mishaps
Travel blogger Cory Lee lives with spinal muscular atrophy (SMA) and travels with his 400-pound power wheelchair. He’s no stranger to travel snafus: his wheelchair charger blew up in Germany, an Alabama fire department rescued him when he was stuck on the top floor of a museum because the elevator broke, and he narrowly escaped being eaten…
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Setback in gene therapy for Duchenne muscular dystrophy as immune system emerges as key barrier
A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.
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Congress Moves Forward on Medicaid Cuts: What You Need to Know
This week, we received concerning news from Washington: Congress has passed a budget resolution that will require significant cuts to Medicaid—up to $880 billion over 10 years. Congress is still determining the exact scope and timeline of these changes, but has taken a step forward to advance them. Below is an update on what this…
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Vamorolone up for approval in Canada as treatment for DMD
Health Canada has agreed to review an application by Kye Pharmaceuticals seeking the approval of vamorolone for treating Duchenne muscular dystrophy (DMD) — and granted it priority review. Priority review shortens the review period from the standard 10 months to six. It’s given to therapies that have the potential to provide significant benefits in treating…
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Watch: Wave Life Sciences – Update on FORWARD-53 Study of WVE-N531 (Webinar Recording)
Wave Life Sciences recently joined PPMD for a community webinar to provide an update on the company’s Phase 2 FORWARD-53 trial of WVE-N531, Wave’s exon skipping oligonucleotide being investigated for the treatment of individuals with Duchenne who are amenable to exon 53 skipping. Wave discussed the recently released FORWARD-53 data and answered questions from the…
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MDA Ambassador Guest Blog: Spreading Awareness One T-shirt at a Time
Dwayne Wilson is 57 years old. He is originally from Southern California and lived in Spokane, WA. for 20 years until moving back home to Irvine, Ca, in 2017. Dwayne was diagnosed with late-onset Pompe disease (LOPD) on Nov 19th, 2018, when he was 50 years old. Dwayne lives with his wife and mother-in law,…
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A Singaporean theater production reveals caregiving complexity
I recently had the opportunity to attend a Singaporean theater production titled “Supervision,” thanks to the SingHealth Patient Advocacy Network (SPAN). SPAN is a collective of patients and caregivers who provide feedback to improve healthcare here in Singapore. I’ve been part of it since 2022. The invitation to the play was a thank-you for volunteering…
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Independent committee favors continuing Elevidys dosing in trials
An independent data monitoring committee (DMC) favors continuing dosing Elevidys (delandistrogene moxeparvovec-rokl) to people with Duchenne muscular dystrophy (DMD) in ongoing clinical trials, according to developer Sarepta Therapeutics. The determination comes after recruitment and dosing in certain clinical trials were paused after a young man who received the gene therapy died due to acute liver…
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In Case You Missed It…
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…
