June 2025

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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CureDuchenne is pleased that one of the main themes at the recent US FDA Cell and Gene Therapy Roundtable was preserving incentives and leveraging regulatory flexibility and innovation to remove obstacles for rare disease treatment. Read more here The post US leadership emphasizes support for rare disease drug development appeared first on CureDuchenne.

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Today, REGENXBIO announced new data from its ongoing Phase I/II AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy being developed for individuals with Duchenne muscular dystrophy. RGX-202 is designed to deliver microdystrophin via AAV8 through a one-time IV infusion. The newly released data comes from boys treated at the higher of two doses (2×1014…

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In this Quest Podcast episode, we chat with a former pharmacist turned singer/songwriter who lives with Generalized Myasthenia Gravis. Dania Quill has devoted her time and expertise to create inclusive spaces for those with disabilities and deliver advice, inspire action, and make us feel closer through song while sharing stories of resilience and positivity.  While…

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PPMD continues to advocate to ensure families navigating a diagnosis of Duchenne and Becker muscular dystrophy have access to optimal care for their loved ones. The Certified Duchenne Care Center Program (CDCC Program), a unique program of PPMD, aims to ensure all individuals with Duchenne and Becker have access to high quality, comprehensive, and coordinated…

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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS Heart’s experimental stem cell therapy for heart problems in Duchenne muscular dystrophy (DMD), the company announced in an email sent to Muscular Dystrophy News Today. This FDA designation aims to accelerate the development of treatments for rare diseases, like DMD,…

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