News

ENVISION Gene Therapy Sarepta Therapeutics, which received early funding from CureDuchenne, has provided a community letter outlining the status of ongoing research in their gene therapy and exon-skipping programs. Of The post Sarepta shares updates with the Duchenne community, including decision to close the ENVISION gene therapy clinical trial appeared first on CureDuchenne.

Satellos Bioscience has announced a change to the primary endpoint in its Phase 2 BASECAMP clinical trial evaluating forazapadin, an investigational oral, small molecule designed to enhance skeletal muscle repair and regeneration in individuals living… The post Satellos Announces Change to Primary Endpoint in BASECAMP Duchenne Trial appeared first on Parent Project Muscular Dystrophy.

“Take care of yourself.” I hear these four little words so often, yet they can be hard to follow. I have seven children with my husband, Jason: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 12; and Callie, 4. Max, Rowen, and Charlie have Duchenne muscular dystrophy (DMD), and I am their The post Back to the basics: My 5-point DMD caregiver self-care plan appeared first on Muscular Dystrophy News.

Satellos Bioscience, which received prior funding from CureDuchenne, has announced that after meeting with the FDA they have amended the primary endpoint in their Phase 2 BASECAMP trial to now The post Satellos Phase 2 trial in boys with Duchenne will use MRI-measured fat fraction as primary efficacy endpoint appeared first on CureDuchenne.

Janna Leach is 43 years old and lives in Louisiana.  She has a bachelor’s degree in education from LSU and currently has my own tutoring business.  Jana loves meeting new people, shopping, football, and spending time with friends and family! I was diagnosed as an infant with spinal muscular atrophy (SMA) in the 1980’s when there… The post MDA Ambassador Guest Blog: A Life Made Possible by Love and Support appeared first on Quest | Muscular Dystrophy Association.

Today, Sarepta Therapeutics shared an update through a community letter regarding its research and clinical development programs, including an important decision about the Phase 3 ENVISION clinical trial evaluating ELEVIDYS in non-ambulatory and late-ambulatory individuals… The post Sarepta Shares Update on ENVISION Clinical Trial and Ongoing Duchenne Research appeared first on Parent Project Muscular Dystrophy.

Treatment with the experimental gene therapy BB-301 was found to improve swallowing ability for every person with a rare type of muscular dystrophy (MD) that mainly affects the throat and eyes who are taking part in a first-in-human clinical trial. That’s according to new interim data announced by Benitec Biopharma, a U.S.-based company developing BB-301 for The post Gene therapy may ease swallowing problems in those with rare MD type appeared first on Muscular Dystrophy News.

We are celebrating a historic achievement for the Race to End Duchenne endurance fundraising program following the 2026 TCS New York City Marathon this past weekend. This year’s team raised $300,000, marking the most successful… The post PPMD’s 2026 NYC Marathon Team Sets Fundraising Record, Going the Distance to Raise Awareness and Funds for Duchenne and Becker Community appeared first on Parent Project Muscular Dystrophy.

I have an ambitious bucket list for someone who needs help with almost everything physically. I want to watch Chelsea play at Stamford Bridge, their home stadium in West London, preferably on a Champions League night. I’d love to write my memoir and pursue a master’s degree in economics, systems design, or thanatology, the study The post Making plans for a future that DMD makes unpredictable appeared first on Muscular Dystrophy News.

Servier announced yesterday that the pivotal Phase 3 GRAND CANYON study evaluating sevasemten in individuals living with Becker muscular dystrophy did not meet its primary endpoint. Sevasemten is an orally administered small molecule inhibitor designed… The post Servier Announces Topline Results from Pivotal Sevasemten Study in Becker appeared first on Parent Project Muscular Dystrophy.

Scientists have discovered a compound that transforms a natural muscle-repair protein into a more powerful version dubbed “Super HGF,” more than doubling its ability to bind to a key receptor while protecting it from age-related chemical damage. The breakthrough, supported by early mouse experiments, could eventually help combat muscle loss and preserve strength as people age.

Becker muscular dystrophy Servier announced that topline results from the 18-month placebo controlled GRAND CANYON study in Becker muscular dystrophy did not meet the primary endpoint, with no statistically significant The post Servier shared that sevasemten did not meet the primary endpoint in the Phase 3 trial in Becker muscular dystrophy appeared first on CureDuchenne.

Valosin-containing protein (VCP)-associated multisystem proteinopathy (VCP-MSP), also sometimes called VCP disease, is a rare inherited condition caused by changes in the VCP gene. These gene changes can affect multiple body systems, resulting in a range of clinical features. Historically, VCP-MSP was known as inclusion body myopathy associated with Paget disease of bone and frontotemporal dementia… The post Simply Stated: Understanding VCP-Associated Multisystem Proteinopathy (VCP-MSP) appeared first on Quest | Muscular Dystrophy Association.

Since I was 6 years old, I’ve been living with the impact of having Duchenne muscular dystrophy. When I was a kid, I loved building with Legos. I liked taking things apart and figuring out how to put them back together. If there were electronics in the house, I was disassembling them and hoping they’d The post Guest Voice: When limitations get in the way, I turn to knowledge and innovation appeared first on Muscular Dystrophy News.

The Muscular Dystrophy Association (MDA) is hailing a recent U.S. federal court ruling that blocks, at least for now, the implementation of new restrictions that would have made it harder for many people with disabling conditions such as muscular dystrophy to qualify for Medicaid, the government-run program that provides health insurance to disabled and low-income The post MDA applauds new US federal court decision on Medicaid coverage appeared first on Muscular Dystrophy News.

Being Heumann opens in selected US cinemas on November 6 and on Apple TV on November 13. The film, from Siân Heder, the Oscar-winning director of CODA (2021), recounts the 1977 Section 504 sit-in, when disability activists occupied the San Francisco offices of the Department of Health, Education and Welfare for 26 days – one… The post Being Heumann: My Unpublished Interview with the Disability Rights Pioneer appeared first on Quest | Muscular Dystrophy Association.

Two interconnected themes — community and hope — dominated the conversations at a recent gathering of people living with neuromuscular diseases. Lily Sander, national ambassador for the Muscular Dystrophy Association (MDA), hit on those themes in the opening talk at MDA Engage: Dallas, an Oct. 23-24 symposium that brought together people living with neuromuscular diseases, The post MDA Engage: Event sessions highlight community, hope appeared first on Muscular Dystrophy News.

One of the greatest experiences of my life is raising children and watching them grow. My husband, Jason, and I share seven children: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 12; and Callie, 4. I have witnessed many beautiful moments in my parenting journey. In my columns, I share my family’s The post The benefits of my son becoming a caregiver for his brothers with DMD appeared first on Muscular Dystrophy News.

The first step to figuring out the right equipment for someone with a neuromuscular disease like muscular dystrophy is to understand that person’s goals, environment, and changing needs, a pair of experts said. “There’s a lot of equipment out there,” said Katie Metheny, an occupational therapist based in the Dallas area. “A lot of folks The post MDA Engage: The right assistive equipment can aid independence appeared first on Muscular Dystrophy News.

Researchers at PTC Therapeutics are conducting the phase 3 Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich’s Ataxia (PROVE-FA) study to learn more about Friedreich’s ataxia (FA) and to evaluate vatiquinone, an investigational therapy being studied for the treatment of FA. About the PROVE-FA study The PROVE-FA study is evaluating the efficacy and safety… The post Clinical Research Alert: Phase 3 Study of Vatiquinone in Individuals with Friedreich’s Ataxia appeared first on Quest | Muscular Dystrophy Association.

I’ve written previously about how those of us with facioscapulohumeral muscular dystrophy (FSHD) often adapt without even thinking about it to increasing weakness as a result of our disease progression. For me, the most recent example of this is the knee grab exercise. This may be an old, well-used tactic for many in the disability The post I found a new FSHD life hack to respond to my worsening leg weakness appeared first on Muscular Dystrophy News.

Scientists have uncovered a hidden defense mechanism in aging muscles that sacrifices strength to protect cells from damage. In mice, partially restoring a crucial molecule reversed signs of muscle wasting and prevented premature death, raising hopes for new treatments for age-related muscle decline.

The 2024 Wheelchair Rule was passed to improve air travel for people with disabilities under the ACAA. But some rules are paused. Here’s what you need to know. The post Some New Accessible Air Travel Rules Are Paused: Here’s What That Means for Travelers with Disabilities appeared first on Quest | Muscular Dystrophy Association.

A new gene therapy approach safely restored the production of emerin, the protein missing in people with X-linked Emery-Dreifuss muscular dystrophy (EDMD), in both a mouse model and human muscle cells, according to a study. Although the preclinical study could not assess the gene therapy’s impact on muscle function, it provided early proof-of-concept data supporting The post Gene therapy restores key protein in models of rare muscular dystrophy appeared first on Muscular Dystrophy News.

As gene therapy research continues to advance, understanding who may be eligible for a particular treatment remains an important challenge for the Duchenne and Becker community. PPMD is committed to supporting research that addresses these… The post Fellowship Update: Universal Antibody Screening Test for AAV Gene Therapy appeared first on Parent Project Muscular Dystrophy.