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Sarepta pauses Duchenne gene therapy for non-ambulatory individuals after second death due to acute liver failure
CureDuchenne is sad to share that another person has passed away due to acute liver failure after treatment with Elevidys, Sarepta’s micro-dystrophin gene therapy. Our hearts go out to the family and friends of this individual at this difficult time. While regulators and experts are reviewing the data and looking at possible safety enhancements including additional…
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MDA Ambassador Guest Blog: How Making an Impact Renewed My Purpose After FSHD Diagnosis
Ranae Beeker, who is in her late 60s, has found her greatest joy in her roles as a mother to Alethea and Gavin, and as a Gramma to Eilif and Zuzu. Ranae’s journey to diagnosis was not without its challenges, as she underwent three misdiagnoses before finally finding answers at Johns Hopkins. where doctors confirmed…
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REGENXBIO WEBINAR
AFFINITY DUCHENNE®: New Phase 1/2 Interim Functional Data Watch HERE The post REGENXBIO WEBINAR appeared first on CureDuchenne.
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Our family’s all-star football player also lives with Duchenne
I’m a mother of seven and a caregiver to the three of my sons who have Duchenne muscular dystrophy (DMD). My oldest daughter, Lexi, 24, lives a few hours away with her husband. I live in rural Nebraska with my husband, Jason, and our six youngest children: Max, 19; Chance, 18; Rowen, 16; Charlie, 14;…
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Duvyzat conditionally approved by EC to treat Duchenne MD
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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PPMD Announces Children’s Hospital of Philadelphia as CDCC, Recipient of Inaugural Clinical Research Designation
PPMD is pleased to announce the expansion of its renowned Certified Duchenne Care Center (CDCC) Program with the certification of the Neuromuscular Program at Children’s Hospital of Philadelphia (CHOP). In a milestone for the program, CHOP becomes not only a CDCC, but also the first center to receive PPMD’s newly established Clinical Research Designation—recognizing excellence…
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After incredible hardships, I still feel blessed
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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Capricor’s Advisory Committee meeting with FDA to be held July 30, 2025
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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Viral vector in LGMD2E gene therapy gets new FDA designation
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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Inclusive Summer Activities
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…
