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Amyotrophic lateral sclerosis (ALS) Phase 3 Clinical Trial: Enrolling PREVAiLS is a phase 3 trial testing an investigational therapy called pridopidine in adults with early, rapidly progressing ALS. The main goal is to evaluate whether pridopidine can slow the disease progression. Inclusion criteria include: Definite or probable ALS diagnosis Between 18 and 80 years old…

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Sharon Townsend spent the first chapter of her adult life focused on creating a loving, comfortable, and happy home for her three children. Now, the 63-year-old has found a new purpose in providing the same warmth and hospitality as a Front Desk Agent at the Hampton Inn. Getting a diagnosis Sharon Townsend Sharon spent the…

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ADSS1 myopathy (formerly known as ADSSL1 myopathy) is a very rare, genetic neuromuscular disease that causes progressive muscle weakness beginning in childhood. It’s a slowly progressive condition that affects skeletal muscles, breathing muscles, swallowing, and, sometimes, the heart. The number of reported cases worldwide ranges from 100 to 200, but many more are believed to…

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Michael Chan, DDS A trip to the dentist can feel overwhelming for many people living with neuromuscular diseases. Muscle weakness, fatigue, swallowing challenges, contractures, and mobility needs can all affect daily oral hygiene and, ultimately, dental visits. But oral health is about much more than having a bright smile — it plays an essential role…

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Having a rare disease is not an easy life. I have limb-girdle muscular dystrophy, so in addition to the physical decline of my body, I also need to be hyperaware of my mental health. I don’t think humans were hardwired to adjust to life on their own with a condition like muscular dystrophy. So what’s…

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Critical ALS Legislation You Need to Know About As we near the end of ALS Awareness Month, the work is just beginning to get critical legislation for those with ALS passed by Congress. This year, MDA is prioritizing and advocating for three major ALS congressional initiatives in service of those living with ALS. First, we…

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An experimental gene therapy called ATA-200 has shown promising safety and efficacy results in an early clinical trial for children with limb-girdle muscular dystrophy type R5 (LGMDR5). Long-term data from the first two treated patients suggest the therapy is working as expected, with no serious side effects, according to developer Atamyo Therapeutics. “These initial results…

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In this episode of the Quest Podcast, we chat with Nicole Lucas, a devoted family caregiver and dental hygienist who stepped away from her career so that her daughter could pursue her dreams; Carly Weber, a 24-year-old law student at the University of Pittsburgh living with spinal muscular atrophy (SMA) who has never let expectations…

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Since her diagnosis with ALS at age 32, Gwen Petersen has poured her energy into advancing the science of her disease through participation in clinical and observational research. Gwen works together with her beloved group, Her ALS Story, to raise awareness that young people can get ALS, too. Gwen is married to her best friend,…

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Living with Duchenne muscular dystrophy (DMD) means your loved one will need to adapt to symptom changes over time. Paying close attention to these changes is key because it can help your loved one maintain a sense of independence and control. It can also help you plan daily life adjustments and prepare for the future.…

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