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Sarepta Therapeutics reported positive data for SRP-5051, their next-generation exon skipping agent designed for individuals amenable to skipping Exon 51. In part B of the Phase 2 MOMENTURM study, ambulatory and non-ambulatory individuals who received the high dose of 30 mg/kg once every 4 weeks had an average dystrophin expression of 5.17% at 28 weeks. This…

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Sarepta Therapeutics, Inc. today announced positive data from Part B of SRP-5051-201, the MOMENTUM study. MOMENTUM is a global, Phase 2, multi-ascending dose clinical trial of SRP-5051 that enrolled patients aged 8 to 21 years. SRP-5051 is a next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for individuals with Duchenne who are amenable to exon 51…

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Last year, PPMD hosted a Duchenne Early Care meeting as part of the 2023 Duchenne Healthcare Professionals Summit, focusing on key aspects of care for the youngest children in our Duchenne community, in preparation for expanding newborn screening for Duchenne. Bringing together experts, the meeting delved into how to best care for babies and toddlers…

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PPMD is excited to announce another significant milestone: Minnesota has officially approved the addition of Duchenne to the state’s newborn screening panel! Approximately 63,000 babies are born in Minnesota every year, which means that we expect this program to identify at least six babies with Duchenne annually once implemented. Minnesota is now the third state,…

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I love to read, but finding time to sit down with a good book can be challenging in my busy house. As a mom to seven and a primary caregiver to my three sons with Duchenne muscular dystrophy (DMD), quiet time is often interrupted by my children. Occasionally, however, I find a book I can’t…

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PPMD is disappointed to learn that the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) today issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarna (ataluren), which will result in the withdrawal of the therapy for Duchenne patients with nonsense mutations  in Europe. The decision…

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The symptoms of my facioscapulohumeral muscular dystrophy (FSHD) make staying positive a daily challenge. Many people with muscular dystrophy struggle with depression. This battle is magnified every winter as I also feel the impact of seasonal affective disorder (SAD). Exposure to sunlight helps our bodies produce vitamin D, a compound that can help boost serotonin…

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As an early funder of PepGen, we are pleased to share that PenGen has dosed the first person in its Phase 2 trial for Duchenne amenable to skipping exon 51.  Initial data , including safety and dystrophin production, at the 5 mg/kg dose is expected mid-20. Read the Press Release HERE The post PepGen Announces…

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AGAMREE (Vamorolone), whose development at ReveraGen Biopharma was supported by CureDuchenne, has been approved in the United Kingdom (UK) for individuals with Duchenne aged 4 years and older. The Medicines Healthcare products Regulatory Agency (MHRA) recognized not just the efficacy of AGAMREE, but also clinical benefits with regards to preserving bone health and maintaining growth…

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We are excited to announce the launch of PPMD’s Gene Therapy Hub! Gene therapy has made significant strides in Duchenne, with one therapy approved and other potential therapies in various stages of development. As our community continues to see progress, many new concepts and terms arise, ones that families have not had to consider before.…

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