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PTC Therapeutics provided an update to the Duchenne community with additional details following the withdrawal of the ataluren New Drug Application (NDA). The update notes that a limited remaining supply of Ataluren is still available, anticipated to last up to six months for individuals currently receiving treatment, and provides details for treating physicians to continue…

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Today PTC Therapeutics released an updated letter to the Duchenne community on their website with additional details following the company’s withdrawal of the ataluren New Drug Application (NDA). This update includes new information on a limited supply of therapy, which PTC anticipates to last up to approximately six months for those currently receiving treatment, as…

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Renowned Napa Valley Vintners and Philanthropists Unite in Newport Beach  for a Weekend of Wine, Food, and Purpose  NEWPORT BEACH, California (March 27, 2026) – CureDuchenne, a global leader in advancing research and care for Duchenne muscular dystrophy, partnered with Vintner Chair Peter Michael Winery to host the 12th annual Napa in Newport: The Premier Wine Weekend at…

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Bridgebio Pharma has submitted an application asking the U.S. Food and Drug Administration (FDA) to approve its experimental oral therapy, BBP-418, to treat limb-girdle muscular dystrophy type 2i (LGMD2i). If approved, BBP-418 would become the first available treatment for LGMD2i, also called LGMDR9. In fact, according to Bridgebio, BBP-418 has the potential to become the…

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Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare neurological disorder in which the immune system mistakenly attacks the protective covering of peripheral nerves. This damage disrupts nerve signaling and can lead to muscle weakness, numbness, and problems with balance and coordination. CIDP is estimated to affect between 1 and 9 people per 100,000 individuals. Cause…

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Mindy Henderson, MDA’s Vice President of Disability Outreach & Empowerment & Editor-in-Chief of Quest Media Today I write to you with a full heart—because there is news that feels nothing short of miraculous. The FDA has approved a higher‐dose regimen of Spinraza (nusinersen)for people living with spinal muscular atrophy (SMA), and I want to share what…

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Living with a rare disease means navigating a healthcare system that often feels fragmented and overwhelming. For many of us, finding a doctor who truly understands our condition is essential. A rare disease specialist does more than treat symptoms. They see the complete picture of who you are and what you face. They understand the…

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As parents, we dedicate our lives to raising our children. We work long and hard, striving to meet their needs, teach kindness, encourage a strong work ethic, and foster self-advocacy. Our goal is to help them grow into successful adults. As a mom of many, I can see my and my husband’s hard work and…

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SRP-1001 and SRP-1003, Sarepta Therapeutics’ investigational RNA-based therapies for two types of muscular dystrophy, were generally well tolerated in early results, according to data from two Phase 1/2 trials. SRP-1001, for facioscapulohumeral muscular dystrophy type 1 (FSHD1), and SRP-1003, for myotonic dystrophy type 1, showed high levels of delivery to muscle in early company-reported data.…

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In this Quest Podcast episode, we chat with advocate, social media influencer, and President of the Periodic Paralysis Association (PPA), Cienna Ditri, who lives with periodic paralysis. Cienna shares her diagnostic journey — from childhood soccer games where something felt “off” to finally getting answers — and how living with an unpredictable condition has shaped…

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