News

PPMD recently hosted a webinar about Fat Embolism Syndrome (FES), covering what FES is, what symptoms to watch for, and how to help keep people with dystrophinopathy safe and healthy. Four clinical experts shared practical… The post Understanding Fat Embolism Syndrome (FES) and Protecting Bone Health appeared first on Parent Project Muscular Dystrophy.

Turning 30 last month was a significant milestone in my Duchenne muscular dystrophy (DMD) journey. For years, my care has revolved around keeping my vital functions strong enough to sustain The post Finding balance as I manage my DMD-associated cardiomyopathy appeared first on Muscular Dystrophy News.

Solid Biosciences Inc. has announced positive new interim data from the Phase 1/2 INSPIRE DUCHENNE clinical trial and provided an update on its planned meeting with the U.S. Food and Drug Administration (FDA) to discuss… The post Solid Biosciences Shares Interim Data from INSPIRE DUCHENNE Trial of SGT-003 appeared first on Parent Project Muscular Dystrophy.

Sarepta Therapeutics has announced top-line results from the ESSENCE trial, a global, randomized double-blind, placebo-controlled Phase 3 confirmatory study evaluating the efficacy and safety of AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen) in individuals living… The post Sarepta Announces Completion of ESSENCE Trial for Exon 45 & 53 Skipping Therapies appeared first on Parent Project Muscular Dystrophy.

PPMD and MyoGene Bio (MyoGene) are excited to announce that PPMD has provided $400,000 in funding to MyoGene through PPMD Venture Pathways, the organization’s venture-philanthropy initiative that provides industry funding to accelerate therapeutic development for… The post PPMD Provides $400,000 in Funding to MyoGene Bio Through PPMD Venture Pathways Program to Support Development of Gene Editing Platform appeared first on Parent Project Muscular Dystrophy.

Expanded access (compassionate use) helps patients access promising treatments when no other options exist. Learn how this process works. The post How Expanded Access and Compassionate Use Broaden Access to Investigational Therapies appeared first on Quest | Muscular Dystrophy Association.

Note: This story was updated Nov. 5, 2025, to clarify that enrollment in the follow-up study is ongoing. Dosing has begun in a long-term follow-up study testing Satellos Bioscience’s experimental The post Oral therapy moves to follow-up after strong data in DMD adults appeared first on Muscular Dystrophy News.

Solid Biosciences today reported an update on the progress of the INSPIRE DUCHENNE open label, Phase I/II clinical trial with SGT-003 and their planned regulatory discussions in 2026. 23 Pediatric The post Solid plans to seek FDA guidance in 2026 for Duchenne Gene Therapy Candidate SGT-003 S appeared first on CureDuchenne.

Sarepta has completed the ESSENCE study, which is a confirmatory trial aimed at supporting full FDA approval of the exon-skipping PMOs Amondys 45 and Vyondys 53.  The study did reinforce The post Sarepta’s Amondys 45 and Vyondys 53 confirmatory trial fails to achieve statistical significance on primary endpoint appeared first on CureDuchenne.

In August, Quest had the privilege of spotlighting published authors living with neuromuscular disease and sharing their books in celebration of National Read A Book Day. With so many talented and accomplished writers in our community and holiday shopping right around the corner, the second installment of our Spotlight on Community Authors blog series is… The post Books to Add to Your Holiday Shopping List This Year: Spotlight on Community Authors appeared first on Quest | Muscular Dystrophy Association.

Last weekend, my husband and I cleaned out our storage unit. My husband loves that unit, while I feel like we need one so that we don’t accumulate too much The post I’m learning to accept that grief is part of my DMD caregiver journey appeared first on Muscular Dystrophy News.

Neuropathy ataxia and retinitis pigmentosa (NARP) syndrome is a rare, maternally-inherited condition caused by mutations in mitochondrial DNA, affecting the energy-producing structures within cells. The condition typically begins in childhood or early adulthood and presents with a wide range of symptoms. These may include learning difficulties, muscle weakness (particularly around the eyes), uncoordinated movements (ataxia),… The post Simply Stated: Updates in Neuropathy Ataxia and Retinitis Pigmentosa (NARP) Syndrome appeared first on Quest | Muscular Dystrophy Association.

Updated 10/31/2015:Letter to the community: October 30, 2025RegenXBio announced that they have completed enrollment in the Phase 1/2 portion of their RGX-202 microdystrophin gene therapy trial, with topline data expected The post RegenXBio completed enrollment in pivotal gene therapy trial;  still enrolling the confirmatory trial ages 1 year and above appeared first on CureDuchenne.

It’s that time of year when we come together to connect, share, learn—while accelerating progress in neuromuscular (NMD) healthcare and scientific progress. This conference is one of the most meaningful gatherings in the (NMD) community—bringing together clinicians, researchers, industry partners, advocates and individuals living with NMD. The four-day conference promises rich content, networking, and collaboration… The post Getting Ready for the 2026 MDA Clinical & Scientific Conference appeared first on Quest | Muscular Dystrophy Association.

In a deal worth roughly $12 billion, pharmaceutical giant Novartis has agreed to acquire Avidity Biosciences, to develop several promising experimental therapies for various types of muscular dystrophy. The core The post Novartis pays $12B for Avidity and its muscle-targeting RNA platform appeared first on Muscular Dystrophy News.

I have a growing list of limitations, as a result of my facioscapulohumeral muscular dystrophy (FSHD), my blindness, and other injuries from a long-ago head-on collision with a drunk driver. The post My family caregivers are feeling the pressure appeared first on Muscular Dystrophy News.

Updated: October 28, 2025 From Belief to Breakthrough: Celebrating Avidity’s Acquisition by Novartis In 2018, CureDuchenne made a bold investment in a promising company with an idea that could change The post Avidity Biosciences to be acquired by Novartis appeared first on CureDuchenne.

Use of the oral therapy BBP-418 appeared to improve walking ability and lung function in people with limb-girdle muscular dystrophy type 2i (LGMD2i) — characterized by muscle wasting at the The post BBP-418 improves walking ability in LGMD2i patients in Phase 3 trial appeared first on Muscular Dystrophy News.

Yesterday, Avidity Biosciences announced a definitive agreement to be acquired by Novartis. The transaction includes Avidity’s late-stage programs, including delpacibart zotadirsen (del-zota) for Duchenne. According to Avidity’s announcement, the company and Novartis intend to keep… The post Avidity Biosciences to be Acquired by Novartis appeared first on Parent Project Muscular Dystrophy.

Funding for the Supplemental Nutrition Assistance Program, which provides food assistance to more than 42 million children and adults across the country – including 4 million people with disabilities – will run out in November as a result of the federal government shutdown. Unless the administration takes emergency measures, like it did with the Special… The post What to Know About Changes to Food Assistance through SNAP appeared first on Quest | Muscular Dystrophy Association.

Italfarmaco issues a letter to address the recent online discussions about the individuals who have passed away while being treated with Duvyzat (givinostat), confirming that they have determined the deaths The post Letter from Italfarmaco about patient deaths while taking givinostat appeared first on CureDuchenne.

Italfarmaco, the company that developed DUVYZAT® (givinostat), has shared a letter to the Duchenne muscular dystrophy community addressing concerns raised in recent online, community-based discussions about patient deaths involving individuals living with Duchenne who were… The post Italfarmaco Addresses Patient Deaths, DUVYZAT Safety Profile in Community Letter appeared first on Parent Project Muscular Dystrophy.

We are on the brink of November, and it’s beginning to turn cold here in Nebraska. My husband and I are debating whether we should turn the heat on. I The post I’m giving everything I have for Duchenne MD — in my own way appeared first on Muscular Dystrophy News.

Data from two clinical trials confirm that Agamree (vamorolone), an approved anti-inflammatory treatment for people with Duchenne muscular dystrophy (DMD), blocks the action of a receptor involved in regulating the The post DMD treatment Agamree may avoid issues of traditional steroids appeared first on Muscular Dystrophy News.

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