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PPMD Joins FDA Leadership for Rare Disease Roundtable
On Wednesday, June 3rd, PPMD was invited to participate in a closed roundtable with senior leadership at the U.S. Food and Drug Administration (FDA). The meeting was convened by Amy Comstock Rick, JD, Director of the Rare Disease Innovation Hub on behalf of Acting Commissioner Kyle Diamantas, JD, Acting Director of CBER, Karim Mikhail, B.…
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Entrada shares updates on Duchenne programs, including initiation of Cohort 2 in UK and EU trial skipping exon 45
As an early funder of Entrada Therapeutics, we are pleased to share their latest newsletter to the Duchenne community. This update includes news that after review of all the data in the first dose (5 mg/kg) cohort of the ELEVATE-45-201 study for individuals amenable to skipping exon 45, an independent data monitoring committee has recommended…
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Adults with MD report major gaps in sexual, reproductive healthcare: Study
Adults with muscular dystrophy (MD) in the U.S. report widespread gaps in sexual and reproductive healthcare, according to surveys and interviews. Such gaps included a lack of private clinical time, provider discomfort, and unaddressed concerns about fertility and relationships. At the same time, participants indicated a strong desire for proactive, disability-informed guidance from their medical…
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Sarepta Webinar
ELEVIDYS Gene Therapy Update and ENDEAVOR Cohort 8 Awareness Watch HERE Recorded: May 19, 2024 The post Sarepta Webinar appeared first on CureDuchenne.
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Tevard Webinar
Unlocking the therapeutic power of suppressor tRNAs for DMD Watch HERE Recorded Thursday, May 28, 2026 The post Tevard Webinar appeared first on CureDuchenne.
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Regenxbio Webinar
RGX-202: Investigational Gene Therapy for Duchenne Muscular Dystrophy Pivotal Data Update from the AFFINITY DUCHENNE® Study Watch HERE Recorded Tuesday, May 19, 2026 The post Regenxbio Webinar appeared first on CureDuchenne.
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Servier’s Acquisition of Edgewise’s Sevasemten Marks Major Milestone for Becker and Duchenne Muscular Dystrophy Communities
The neuromuscular disease community received encouraging news this week as Edgewise Therapeutics announced the sale of sevasemten and its Becker and Duchenne muscular dystrophy programs to Servier, a global pharmaceutical company with a strong track record of developing and delivering innovative therapies to patients worldwide. CureDuchenne invested in Edgewise in 2019, recognizing the potential of their approach. This acquisition is…
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MDA Joins Forces to Protect NIH Funding
It’s no secret that MDA advocates are a force on Capitol Hill and have routinely commanded the hall of Congress during MDA on the Hill over the years. Jennifer Lane and Rep Aderholt But what if MDA teamed up with volunteers and advocates from other organizations to come together as one powerful group? That is…
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Simply Stated: Introduction to Morimoto-Ryu-Malicdan Neuromuscular Syndrome (RFC4 deficiency)
Morimoto–Ryu–Malicdan neuromuscular syndrome (MRMNS) is an inherited condition that was first reported on in late 2024. It is caused by variants in the RFC4 gene and is classified as a congenital myopathy, primarily affecting skeletal muscles and in some cases the nervous system. Much about the symptoms and progression of this newly discovered condition remains…
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Helping my sons with DMD make connections has also helped me
Here are some facts about me: I am an introvert, I am quiet, and my strongest voice is the written word. I grew up in the foster care system and experienced childhood trauma that makes it hard for me to know how to be around people. I am a mom to seven children: Lexi, 25,…
