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The European Comission has conditionally approved Duvyzat (givinostat) to be taken alongside corticosteroids for treating people who are 6 and older with Duchenne muscular dystrophy (DMD). “This milestone means that a broad range of patients with DMD have access to a new treatment,” Francesco De Santis, president of Italfarmaco Holding and chairman of Italfarmaco Group,…

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Capricor Therapeutics, which received early funding from CureDuchenne, announced regulatory updates for their Deramiocel program to treat Duchenne.  The FDA informed Capricor of its intent to hold an Advisory Committee meeting on July 30 of this year.   Capricor‘s manufacturing facility has completed inspection and the company is on track for a priority review with the…

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I’m between two anniversaries. Today is the 36th anniversary of the collision I had with a drunk driver that cost me my eyesight and almost cost me my life. Saturday will be my 45th wedding anniversary. The car crash happened on the evening of June 11, 1989. I was returning home from playing music at…

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The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector used in SRP-9003, an investigational gene therapy for limb-girdle muscular dystrophy type 2E (LGMD2E). “This is one of the first programs to receive platform technology designation and an important recognition by FDA of the reproducibility and adaptability of this technology…

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As every father knows, parenthood is an incredible journey filled with joy, challenges, victories, growth, and love. For dads living with a disability, raising children sometimes requires extra patience, resilience, and adaptability. In beautiful synchronicity, those very traits are often strengthened through daily life with a disability and serve as an asset to parenting, providing…

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As the school year comes to a close, we are all looking forward to a fun-filled summer! We know that planning outings and activities can sometimes feel overwhelming, especially when considering accessibility and necessary accommodations to ensure everyone’s safety and enjoyment. With your valuable ideas and feedback in mind, PPMD has compiled a list of…

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Dyne Therapeutics recently joined PPMD for a community webinar on the DELIVER clinical trial evaluating DYNE-251. The Dyne team provided the latest updates on the Phase 1/2 global DELIVER study of the company’s investigational therapy for individuals living with Duchenne who are amenable to exon 51 skipping. Watch the recording The post Watch: Dyne Therapeutics…

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I believe that many adults living with rare diseases can and do become experts in their conditions, sometimes more so than healthcare professionals. With respect to my own journey with limb-girdle muscular dystrophy, this point became particularly clear when I had a near-death experience in early 2024. Several types of muscular dystrophy (MD) are rare,…

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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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When I think about my son Charlie, I always remember my pregnancy with him. I’d taken a home test and knew I was pregnant, but when I called to make an appointment with my doctor to confirm the good news, I scheduled an appointment for my son Max, who was 4, at the same time.…

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