News

The CITGO Lake Charles Refinery raised more than $750,000 to support the Muscular Dystrophy Association (MDA) at its 41st annual MDA Golf Classic, an annual golf fundraiser. Since 1985, CITGO Lake Charles has raised $8.5 million for the MDA. The total amount raised this year — $756,800 — set a new record. As in past The post CITGO Lake Charles golf classic raises record $756K to support MDA appeared first on Muscular Dystrophy News.

Hello! In this blog, I’m sharing accessible beauty and fashion tips I’ve collected through plenty of trial and error. Beauty and fashion are major forms of self-expression for me, and I know I’m not alone in that. People with disabilities deserve to present themselves to the world in whatever way feels authentic and comfortable to… The post Life with Lily: My Favorite Accessible Fashion & Beauty Tips appeared first on Quest | Muscular Dystrophy Association.

On June 22, 2026, PPMD Chief Executive Officer  Katherine Beaverson, MS attended the U.S. Department of Health and Human Services (HHS) Clinical Trials Roundtable in Washington, D.C., joining leaders from across government, industry, academia, and… The post Inside the HHS Clinical Trials Roundtable: Why Speed, Access, and Rigor Matter for the Duchenne Community appeared first on Parent Project Muscular Dystrophy.

Each year, I attend at least one national gathering of people living with muscular dystrophy. In addition to learning about the most updated care standards, treatment strategies, and research initiatives, I’m able to bond with those who share a similar diagnosis, and have made some dear friends. The upcoming Parent Project Muscular Dystrophy (PPMD) 2026 The post At PPMD, I get to be part of a fraternity that none of us signed up for appeared first on Muscular Dystrophy News.

Some events are hard to picture before you attend. You may wonder who will be there, whether the topics will apply to you, or if it is worth carving out the time. MDA Engage is designed for people living with neuromuscular disease, families, caregivers, and loved ones who want practical information, real conversation, and a… The post Five Things You’ll Find at MDA Engage appeared first on Quest | Muscular Dystrophy Association.

June is a quiet month here in rural Nebraska. Most days, only neighbors drive on the street outside our home, and you can hear the wind moving through the plains. That may sound boring to some of you from the big cities, but here it is supposed to be relaxing and serene. My children have The post The remedy for my early summer caregiver burnout? Having fun. appeared first on Muscular Dystrophy News.

Researchers at Alexion, AstraZeneca Rare Disease are conducting a clinical study for adults with generalized myasthenia gravis (gMG) who are currently receiving ravulizumab treatment. The study will evaluate whether a planned schedule for gradually lowering oral corticosteroids (OCS) can safely help patients reduce or stop these medications while keeping their gMG symptoms stable. The study… The post Clinical Research Alert: Clinical Study for Adults with Generalized Myasthenia Gravis (gMG) appeared first on Quest | Muscular Dystrophy Association.

Health Canada has accepted and granted priority review to Italfarmaco’s application seeking the approval of oral givinostat, sold in the U.S. under the brand name Duvyzat, to treat Duchenne muscular dystrophy (DMD). The priority review status is reserved for therapies that could significantly improve the benefit-risk profile over current treatments. This designation shortens the evaluation The post Health Canada speeds up its review of givinostat for Duchenne appeared first on Muscular Dystrophy News.

Rob Roozeboom is the founder of RISE Ministries and the creator of RiseFest, one of the largest Christian music festivals in the Midwest. Living in Iowa with muscular dystrophy, Rob is passionate about sharing hope, encouraging others through faith, and advocating for people living with disabilities. He and his wife are proud parents who believe… The post MDA Ambassador Guest Blog: Lessons in Life and Fatherhood appeared first on Quest | Muscular Dystrophy Association.

Back in March, I wrote about how living with Duchenne muscular dystrophy (DMD) has shaped my perspective on accessible design. After decades of moving through systems that were not built with people like me in mind, I had come to appreciate how disability can offer valuable insights into solving accessibility challenges. A couple of months The post Collaboration is imperative to accessible design appeared first on Muscular Dystrophy News.

The experimental medication delpacibart braxlosiran (del-brax) reduced biological markers of facioscapulohumeral muscular dystrophy (FSHD) disease activity in a Phase 1/2 clinical trial, meeting the study’s goals, according to developer Novartis. Based on results from earlier parts of the trial, FORTITUDE (NCT05747924), investigators selected a dosage for this group of participants, dubbed the biomarker cohort. Intravenous The post Experimental drug del-brax curbs signs of muscle damage in FSHD: Study appeared first on Muscular Dystrophy News.

Skye Anderson lives in North Carolina, and is an advocate living with facioscapulohumeral muscular dystrophy (FSHD). She is passionate about accessibility, spreading awareness, and empowering others with disabilities to use their voice. In Skye’s free time, she enjoys expressing herself through art, cheering on the Carolina Hurricanes, and connecting with others through shared experiences. I… The post MDA Ambassador Guest Blog: Strong Enough to Carry Grief AND Gratitude with FSHD appeared first on Quest | Muscular Dystrophy Association.

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to GEn1E Lifesciences‘ GEn-1123 as a potential treatment for Duchenne muscular dystrophy (DMD). The designations aim to incentivize the development of treatments for rare disorders, which are those affecting fewer than 200,000 people in the U.S. Orphan drug status provides The post FDA grants orphan drug, rare pediatric disease status to GEn-1123 for DMD appeared first on Muscular Dystrophy News.

For the neuromuscular disease community, the ADA provides protections for people with disabilities to work and participate fully in their communities. The post MDA’s Guide to the Americans with Disabilities Act (ADA) appeared first on Quest | Muscular Dystrophy Association.

If you read my last column, you’ll know that I’ve been experiencing extreme physical challenges in the last couple months. These changes are a result of a combination of my facioscapulohumeral muscular dystrophy, damage from a major automobile accident many years ago, and simply growing old. This has resulted in a lot more pressure on The post Adding new adaptations around the home to allow my caregivers a break 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.

Among women who carry mutations that can cause Becker muscular dystrophy (BMD) or Duchenne muscular dystrophy (DMD), measures of heart health — important given that heart muscle damage is a key driver of death in most people with these muscular dystrophy (MD) types — are generally stable over time. Those are the findings of a The post Stable heart health seen long-term for women with BMD, DMD mutations appeared first on Muscular Dystrophy News.

Daniel Guillen, MD, explains how DMD affects the heart muscle and why cardiac disease often progresses quietly over time. The post Cardiac disease in Duchenne appeared first on Muscular Dystrophy News.

As of today, Massachusetts has officially begun screening all newborns for Duchenne muscular dystrophy. This milestone comes after the passage of the state’s Maternal Health bill (H. 4999), which was signed into law by Governor… The post Massachusetts Begins Newborn Screening for Duchenne appeared first on Parent Project Muscular Dystrophy.

On Wednesday, June 3rd, PPMD was invited to participate in a closed roundtable with senior leadership at the U.S. Food and Drug Administration (FDA). The meeting was convened by Amy Comstock Rick, JD, Director of… The post PPMD Joins FDA Leadership for Rare Disease Roundtable appeared first on Parent Project Muscular Dystrophy.

Tina Vassar lives in Durham, North Carolina with her dog Milo. She is 69 years young and has lived with myasthenia gravis for over 30 years. Like many people living with myasthenia gravis, I have a unique story – or, as I call it, my voyage. I was diagnosed with anti-ACHR+ generalized myasthenia gravis (gMG)… The post MDA Ambassador Guest Blog: A Myasthenia Gravis Voyage of Twists, Turns, and Discoveries appeared first on Quest | Muscular Dystrophy Association.

It’s no secret that MDA advocates are a force on Capitol Hill and have routinely commanded the hall of Congress during MDA on the Hill over the years. But what if MDA teamed up with volunteers and advocates from other organizations to come together as one powerful group? That is exactly what happened last week… The post MDA Joins Forces to Protect NIH Funding appeared first on Quest | Muscular Dystrophy Association.

Researchers at Vor Bio are conducting a global phase 3 clinical study (UPSTREAM MG) for adults with generalized myasthenia gravis (gMG) who are AChR or MuSK+. The study will evaluate whether treatment with the investigational drug telitacicept can reduce the level of disease-causing B cells and autoantibodies, and improve muscle weakness and fatigue, in people… The post Clinical Research Alert: Clinical Study for Adults with Generalized Myasthenia Gravis (gMG) appeared first on Quest | Muscular Dystrophy Association.

Morimoto–Ryu–Malicdan neuromuscular syndrome (MRMNS) is an inherited condition that was first reported on in late 2024. It is caused by variants in the RFC4 gene and is classified as a congenital myopathy, primarily affecting skeletal muscles and in some cases the nervous system. Much about the symptoms and progression of this newly discovered condition remains… The post Simply Stated: Introduction to Morimoto-Ryu-Malicdan Neuromuscular Syndrome (RFC4 deficiency) appeared first on Quest | Muscular Dystrophy Association.

The MDA Quest Media Product Guide is designed to help you find the products you need to live a more independent, stylish, fun, and all-around great life. All the products you see here were chosen by MDA Ambassadors, who shared exactly how each product helps them in their daily lives. Find something for yourself, or… The post A Look Back at the First Quest Product Guide of 2026 appeared first on Quest | Muscular Dystrophy Association.