News

Here in the Midwest, June brings warmer temperatures. I live in Nebraska, where warmer weather is welcome after bitterly cold winters and brisk, windy springs. It’s also necessary for the The post Finding balance while parenting children with DMD can be tricky appeared first on Muscular Dystrophy News.

Genetic counselors answer frequent questions about genetic testing: How much does genetic testing cost? Should you be tested? The post Your Guide to Genetic Testing for Neuromuscular Diseases appeared first on Quest | Muscular Dystrophy Association.

Our recent investment in Entos Pharmaceuticals (https://entospharma.com) aims to explore the potential of their non-viral, redosable Fusogenix PLV platform to deliver full-length dystrophin protein to all muscle groups in a The post CureDuchenne’s latest investment aims to address the limitations of current AAV-delivered gene therapy treatments for Duchenne muscular dystrophy (DMD). appeared first on CureDuchenne.

Living with Duchenne muscular dystrophy (DMD) has taught me countless lessons, especially the power of connection, creativity, and celebrating every form of love. Last Saturday, I attended the 11th annual The post Finding harmony and inclusion in life through music appeared first on Muscular Dystrophy News.

A second person with Duchenne muscular dystrophy (DMD) has died of acute liver failure after being treated with the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl). A first death was announced The post Elevidys halted in nonambulatory DMD after second death reported appeared first on Muscular Dystrophy News.

It’s the most wonderful time of the year! The holiday’s season already? No! Summertime! That’s right! The time of year when the days are long and hot, the nights are warm and filled with fireflies, and the atmosphere is prime for active living. And for those in the neuromuscular disease community, summertime is the perfect… The post Insights by Ira: Let’s Get Active This Summer! appeared first on Quest | Muscular Dystrophy Association.

Letter to the Community: The post Sarepta pauses Duchenne gene therapy for non-ambulatory individuals after second death due to acute liver failure appeared first on CureDuchenne.

We are heartbroken to learn of the loss of another member of our community following treatment with ELEVIDYS. According to Sarepta, this represents the second reported case of acute liver failure in a non-ambulatory patient… The post A Safety Update on ELEVIDYS – June 2025 appeared first on Parent Project Muscular Dystrophy.

AFFINITY DUCHENNE®: New Phase 1/2 Interim Functional Data Watch HERE The post REGENXBIO WEBINAR appeared first on CureDuchenne.

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 The post Our family’s all-star football player also lives with Duchenne appeared first on Muscular Dystrophy News.

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… The post MDA Ambassador Guest Blog: How Making an Impact Renewed My Purpose After FSHD Diagnosis appeared first on Quest | Muscular Dystrophy Association.

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,… The post PPMD Announces Children’s Hospital of Philadelphia as CDCC, Recipient of Inaugural Clinical Research Designation appeared first on Parent Project Muscular Dystrophy.

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 The post Duvyzat conditionally approved by EC to treat Duchenne MD appeared first on Muscular Dystrophy News.

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 The post Capricor’s Advisory Committee meeting with FDA to be held July 30, 2025 appeared first on CureDuchenne.

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 The post After incredible hardships, I still feel blessed appeared first on Muscular Dystrophy News.

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… The post Watch: Dyne Therapeutics — The Clinical Impact of the FORCE™ Platform for Duchenne (Webinar Recording) appeared first on Parent Project Muscular Dystrophy.

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… The post The Joys (and Lessons) of Fatherhood from MDA Ambassadors appeared first on Quest | Muscular Dystrophy Association.

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… The post Inclusive Summer Activities appeared first on Parent Project Muscular Dystrophy.

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 The post Viral vector in LGMD2E gene therapy gets new FDA designation appeared first on Muscular Dystrophy News.

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 The post We’re our own best experts, but what if we’re incapacitated? appeared first on Muscular Dystrophy News.

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… The post In Case You Missed It… appeared first on Quest | Muscular Dystrophy Association.

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 The post US leadership emphasizes support for rare disease drug development appeared first on CureDuchenne.

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… The post PPMD’s Certified Care Center Program appeared first on Parent Project Muscular Dystrophy.

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 The post Fear and pride as my son starts up with high school band appeared first on Muscular Dystrophy News.

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… The post Quest Podcast: Invisible People: Making the Rare Seen appeared first on Quest | Muscular Dystrophy Association.