My grandson Shepherd has been living with his Duchenne diagnosis for 5 years. Since that day in 2019, our family has turned into crusaders for the Cause.  It has been my privilege and honor to use every trick, tool, and trade God has given me to raise awareness and money for a cure. The post Miner Moebel Family appeared first on CureDuchenne ..read more

Our Duchenne journey began like so many others. Gabe, our fourth son, wasn’t reaching his developmental milestones. His doctor told us there was nothing wrong with him, and that, as the “baby,” he was just spoiled. When we received his diagnosis, we heard that phrase that makes time stand still: “Give him a good life, because it will be a short one.” Gabe was diagnosed just days before his first birthday. Like so many other Duchenne parents, we began researching the best doctors, clinical trials, etc., and began doing as much as we could to give our son the best, longest life possible. Not too ..read more

While many individuals who use mobility devices may be interested in driving, the journey to a wheelchair-accessible vehicle often involves navigating significant physical and financial hurdles.   Accessible vehicles and adaptive technologies offer independence but come with high costs—sometimes as much as $80,000 – according to the National Highway Traffic Safety Administration. Starting the process early is crucial, as it involves several steps to identify the right vehicle and secure necessary funding through organizations and government aids.  From Idea to Reality Race Martinez ..read more

This past year the CureDuchenne Clinic at the Neurology & Neuromuscular Care Center, led by Dr. Diana Castro in Denton, TX, has been marked by unparalleled advancements, notably with the administration of the first gene therapy for Duchenne muscular dystrophy. The CureDuchenne Clinic has shown the power of care — not just medical care — but the personal care of the doctors and clinicians who empower patients with Duchenne to live a high-quality life. These past 12 months have been a testament to that power.  The CureDuchenne Clinic offers care to uninsured and underinsured individuals ..read more

Capricor Therapeutics, which received early funding from CureDuchenne, has shared news of positive interactions with the FDA for their CAP-1002 program for DMD.  Capricor will be having an additional meeting with the FDA in Q2 2024, where they will share data and discuss the plan for submitting a rolling BLA, which might expedite their path to potential drug approval.  Read the Full Press Release HERE 2024-04-24_Capricor_Therapeutics_Announces_Positive_Type_B_275Download The post Capricor Therapeutics Announces Positive Type-B Meeting with FDA for CAP-1002 Program for Duchenne Musc ..read more

Dealing with a fracture is a frightening experience for anyone, especially someone living with Duchenne muscular dystrophy, who has weaker bones and is more prone to falling.   Often, you will have to work with emergency room staff who aren’t as familiar with Duchenne and don’t know the signs of fatty embolism syndrome (FES). Then you, along with an orthopedic doctor, will need to decide whether to surgically or non-surgically fix the fracture.   Because an emergency like this can happen in an instant, we want to help you be prepared. Remember the acronym, “R.A.R.E”: Ready ..read more

Southern California’s Premier Wine Event Brought Together Acclaimed Napa Valley Vintners and Philanthropists to Drive Research for Duchenne Muscular Dystrophy NEWPORT BEACH Calif., April 24, 2024 – CureDuchenne and Vintner Chair Alpha Omega hosted the Tenth Annual Napa in Newport on Saturday, April 20, 2024, at Pendry Newport Beach, which raised more than $1.35 million to find and fund a cure for Duchenne muscular dystrophy, a progressive neuromuscular disease. CureDuchenne is a global leader in research, patient care and innovation for improving and extendin ..read more

Edgewise Therapeutics Announces Positive Two-Year Topline Results from the ARCH Open Label Trial of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy (Becker) – The North Star Ambulatory Assessment (NSAA) remained stable relative to declines reported in Becker natural history studies – – Significant decreases were observed in circulating levels of creatine kinase (CK) and fast skeletal muscle troponin I (TNNI2), biomarkers associated with skeletal muscle damage – – Sevasemten was well-tolerated – – Edgewise leadership to discuss ARCH findings on Tuesday, April 16 at 8:30 a.m. East ..read more

 “Disco For Duchenne” Gala Featured Dancing, Drinks, a Culinary Experience and More   to Help Advance Research for the Rare Disease  AUSTIN, Texas., April 16, 2024 – CureDuchenne, a leading global nonprofit focused on finding and funding a cure for Duchenne muscular dystrophy, and the Revell family of Austin hosted the 15th annual fundraiser gala, Champions to CureDuchenne: Disco for Duchenne, on April 6, 2024. The event raised $385,000 to benefit CureDuchenne as it accelerates scientific research and pioneers educational programs to care for families affected by Duchenne, a pro ..read more

Champion Volunteer Job Board  Welcome to the CureDuchenne Champions Volunteer Opportunities board! Here, you can explore a range of rewarding volunteer positions dedicated to supporting individuals and families affected by Duchenne muscular dystrophy. Whether you’re passionate about fundraising, advocacy, event planning, or community outreach, there’s a role for you to make a meaningful difference in the lives of those living with Duchenne. Join us in our mission to accelerate research, raise awareness, and provide vital resources. Browse our listings and become a Champion for a Cure toda ..read more