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FDA authorizes a new liver safety biomarker relevant for Duchenne and Becker
We congratulate C-PATH (Critical Path Institute) on their success in getting the FDA to qualify a new biomarker– glutamate dehydrogenase (GLDH)– to monitor drug-induced liver injury in clinical trials. This development is important in Duchenne and Becker because other biomarkers typically used to monitor liver injury (such as alanine aminotransferase, or ALT) can already be…
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New FDA Elevidys label limits use to DMD patients who can walk
The U.S. Food and Drug Administration (FDA) has approved an update to the prescribing information for Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec-rokl), removing its conditional approval for DMD patients who can’t walk and including new safety warnings and precautions in light of two deaths related to the therapy reported earlier this year. “We want…
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5 Things Advocates Should Know: The Alleviating Barriers for Caregivers Act
November is National Family Caregivers Month, and it is important to MDA to uplift the importance of family caregivers in the neuromuscular community. Due to decades of underinvestment in paid care, and with welcome breakthroughs in research leading to longer lives for many, the often-invisible frontline of family caregivers is under increasing pressure as the…
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Insmed’s ASCEND Study and INS1201, an Investigational Gene Therapy for Duchenne Muscular Dystrophy
Watch Webinar HERE The post Insmed’s ASCEND Study and INS1201, an Investigational Gene Therapy for Duchenne Muscular Dystrophy appeared first on CureDuchenne.
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Progress Now: Immunotherapy Study Recruiting, Drug Cleared for Expanded Access, and More
Amyotrophic lateral sclerosis (ALS) Immunotherapy Study Recruiting Coya Therapeutics has launched the phase 2 ALSTARS trial to evaluate the safety and efficacy of COYA 302, an investigational treatment for adults with ALS. This large trial will recruit an estimated 120 people with ALS and be conducted at approximately 25 sites in the US and Canada.…
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New Year, New Financial Plan: Tips to Get Your Budget Back On Track
For many families living with neuromuscular diseases, balancing health and disability-related expenses with rising household costs can feel like an impossible feat. Jody Ellis Fortunately, the new year is the perfect time to reassess your financial goals and start with a fresh plan. Here, MDA community members and experts offer their tips to avoid debt,…
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Understanding Spinal-Bulbar Muscular Atrophy
Spinal-bulbar muscular atrophy (SBMA) is a genetic disorder that affects muscles and nerves, leading to loss of nerve cells in the spinal cord and brainstem. The “bulbar” part of SBMA refers to the bulb-like structure in the lower part of the brain, which controls the muscles in the face, mouth, and throat. People with SBMA…
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From Classroom to Courtroom: How Eric Arnold Navigated Accessibility in His Education and Career
Eric Arnold has built his life around sharing his skills and expertise with others — and embracing opportunities to show the world what he brings to the table. The 39-year-old, West Virginia-based attorney attributes his success to recognizing his own capabilities, working hard to reach his potential, connecting with others, and asking for accommodations. Eric…
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A Lifetime of Learning: From college to a Career in Disability Advocacy
I’ve always known that I love to learn. That passion has taken me places I never expected and set me on a path toward making a difference in advancing disability rights. Thriving in college When I was in high school in Clarksville, Maryland, I knew I wanted to attend a four-year college, and I worked…
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The week we ran out of our emotional ‘leftovers’
When I was a young mom watching my oldest daughter, Lexi, move from upper elementary to middle school, I often felt heartbroken and helpless. She was an excellent student and friend at school, but when she came home, her exhaustion could turn into crankiness, and it felt like she was shutting me out. A friend…
