News

PPMD and Secretome Therapeutics (Secretome) are excited to announce that PPMD has provided $250,000 in funding to Secretome through PPMD Venture Pathways, our venture-philanthropy initiative that provides funding to industry to accelerate therapeutic development for… The post PPMD Provides $250,000 in Funding to Secretome Therapeutics Through PPMD Venture Pathways Program to Support Development of Cardiac Cell Therapy appeared first on Parent Project Muscular Dystrophy.

Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant full approval for Amondys 45 (casimersen) and Vyondys 53 (golodirsen), two exon-skipping therapies designed to treat certain people with Duchenne muscular dystrophy (DMD). The company said it met with the FDA to discuss plans for the two therapies, and the FDA The post Sarepta plans to seek full FDA approval of 2 Duchenne treatments appeared first on Muscular Dystrophy News.

According to the EveryLife Foundation for Rare Diseases, more than 30 million Americans live with at least one rare disease. Considering that’s nearly 10% of the U.S. population, these diseases don’t seem so rare after all. For decades, the disability rights movement has fought for the rights of this population, advocating for greater accessibility and The post When it comes to rare disease advocacy, just keep showing up appeared first on Muscular Dystrophy News.

Muscular Dystrophy News Today is wrapping up coverage of the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which ran from March 8-11 in Orlando, Florida, and online. This year, more than 2,400 attendees from 40 countries gathered in person and virtually to discuss recent advances in neuromuscular disease research and care — and The post MDA 2026: This year’s MDA meeting provided a glimpse into the future appeared first on Muscular Dystrophy News.

The comment comes in many forms: “I don’t know how you do it.” “You have your hands full!” “I can’t imagine what it would be like.” It’s always in response to seeing my large family. I share seven children with my husband, Jason: Lexi, 24; Max, 20; Chance, 18; Rowen, 17; Charlie, 15; Mary, 11; The post I’m thankful for a big family that gives my DMD sons a full social life appeared first on Muscular Dystrophy News.

Dyne Therapeutics is advancing its investigational exon-skipping therapy zeleciment rostudirsen (z-rostudirsen), formerly known as DYNE-251, toward regulatory approval after trial data showed early signs of benefit across multiple disease measures in boys with Duchenne muscular dystrophy (DMD). The most recent data from the Phase 1/2 DELIVER clinical trial (NCT05524883) showed that the therapy increased levels of The post MDA 2026: Dyne advances DMD therapy z-rostudirsen toward approval appeared first on Muscular Dystrophy News.

Vocational training, technical education, or a trade school may be even more affordable than you think with these resources and support. The post How to Make Vocational Training Affordable appeared first on Quest | Muscular Dystrophy Association.

In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To cope with all the changes in her life, The post 17-year-old advocate shares her NMOSD story to raise awareness appeared first on Muscular Dystrophy News.

Today, Sarepta Therapeutics announced plans to submit supplemental New Drug Applications (sNDAs) to the U.S. Food and Drug Administration (FDA) for AMONDYS 45 and VYONDYS 53, the company’s exon-skipping therapies for the treatment of individuals… The post Sarepta Announces Plans to Submit sNDAs for AMONDYS 45 and VYONDYS 53 appeared first on Parent Project Muscular Dystrophy.

Boys with Duchenne muscular dystrophy (DMD) who received Sarepta Therapeutics’ gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial continue to show signs of slowed disease progression relative to DMD’s natural course, along with caregiver-reported improvements in everyday living. That’s according to data from the Phase 3 EMBARK clinical trial (NCT05096221) presented at the 2026 The post MDA 2026: ‘Body of evidence for Elevidys continues to grow’ appeared first on Muscular Dystrophy News.

For most of my life, Duchenne muscular dystrophy has defined how I navigate the world. Duchenne is a genetic condition that causes progressive muscle loss. Over time, it weakens the arms, legs, breathing muscles, and heart. Many adults with Duchenne, including me, use wheelchairs and rely on a ventilator to breathe. Daily life often involves The post Living with Duchenne grants me unique insight into accessible design appeared first on Muscular Dystrophy News.

The latest Phase 3 trial results continue showing that deramiocel, an investigational cell therapy for heart disease related to Duchenne muscular dystrophy (DMD), significantly slows the progression of arm and heart damage in boys and men with DMD. Based on the positive Phase 3 results, Capricor Therapeutics has resubmitted an application for U.S. approval of The post MDA 2026: FDA decision on deramiocel for DMD expected by August appeared first on Muscular Dystrophy News.

Sarepta Therapeutics, Inc. has announced screening and enrollment are underway in Cohort 8 of the company’s ENDEAVOR study. ENDEAVOR is an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS in multiple cohorts… The post Sarepta Announces Initiation of Screening and Enrollment for ENDEAVOR Cohort 8 in Non-Ambulatory Individuals Living with Duchenne appeared first on Parent Project Muscular Dystrophy.

Myotonic dystrophy type 1 (DM1) patients in a Phase 1/2 clinical trial saw gains in motor function and cognitive measures after receiving Dyne Therapeutics’ zeleciment basivarsen (z-basivarsen), previously known as DYNE-101, supporting the recent initiation of a Phase 3 study. Findings from the Phase 1/2 ACHIEVE trial (NCT05481879) and the design of the HARMONIA Phase The post MDA 2026: Dyne therapy boosts strength, cognition in DM1 appeared first on Muscular Dystrophy News.

Richard Farrell Jr. is 19 years old and lives in Pennsylvania. He was diagnosed with Becker muscular dystrophy (BMD) when he was 5 years old. He loves to repair computers, play musical instruments, play video games, and talk with his friends. Living with a disability has required me and my parents to rise to meet… The post MDA Ambassador Guest Blog: Navigating a Busy Life with a Feeding Tube appeared first on Quest | Muscular Dystrophy Association.

Each year, the Duchenne and Becker muscular dystrophy community comes together in Washington, DC with a shared mission: to ensure that the voices of individuals living with Duchenne and Becker and their families are heard… The post Community Comes Together on Capitol Hill: A Recap of PPMD’s 2026 Advocacy Conference appeared first on Parent Project Muscular Dystrophy.

Capricor Therapeutics has announced additional analyses and new functional outcomes data from the Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne. Deramiocel is the company’s investigational cell therapy for the treatment of Duchenne cardiomyopathy…. The post Capricor Therapeutics Announces Positive Data from HOPE-3 Study of Deramiocel appeared first on Parent Project Muscular Dystrophy.

Solid Biosciences Inc. has shared updated positive interim data from the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin… The post Solid Biosciences Shares Interim Positive Update on Phase 1/2 INSPIRE DUCHENNE Trial appeared first on Parent Project Muscular Dystrophy.

REGENXBIO has announced new positive interim data from the 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… The post REGENXBIO Shares Positive Interim Data from Phase I/II AFFINITY DUCHENNE Trial of RGX-202 appeared first on Parent Project Muscular Dystrophy.

People from across the neuromuscular field gather to learn and collaborate at the 2026 MDA Clinical & Scientific Conference. The post 2026 MDA Clinical & Scientific Conference Opening Highlights the Power of Collaboration appeared first on Quest | Muscular Dystrophy Association.

Edgewise Therapeutics, Inc. has shared long-term data from its MESA open-label extension study of sevasemten in Becker muscular dystrophy. Sevasemten is an orally administered small molecule inhibitor designed to protect muscle against contraction-induced damage in… The post Edgewise Announces Positive Long-Term Sevasemten Data appeared first on Parent Project Muscular Dystrophy.

Capricor Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has lifted the July 2025 Complete Response Letter and resumed review of the company’s Biologics License Application (BLA) for Deramiocel, an investigational cell… The post Capricor Therapeutics Announces New PDUFA Date for Deramiocel appeared first on Parent Project Muscular Dystrophy.

PPMD, in partnership with the Foundation to Eradicate Duchenne (FED), is pleased to announce Representative Troy Balderson (OH-12), Senator Susan Collins (ME), Senator Amy Klobuchar (MN), Representative Doris Matsui (CA-07), and Senator Roger Wicker (MS)… The post PPMD and FED Announce 2026 Advocacy Leadership Awards Honoring Bipartisan Champions in Congress appeared first on Parent Project Muscular Dystrophy.

Researchers at the University of Minnesota are seeking boys with Duchenne muscular dystrophy (DMD) to participate in a phase 1 clinical trial to evaluate the safety and efficacy of the investigational cell-based therapy MyoPAXon in combination with the immunosuppressant therapy tacrolimus. The current study is examining whether this investigational treatment is safe and well-tolerated in people… The post Clinical Study Alert: Phase 1 Study of MyoPAXon in Boys with DMD appeared first on Quest | Muscular Dystrophy Association.

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.