Atamyo Therapeutics is seeking approvals in Europe to launch its first trial testing ATA-100, its investigational gene therapy for a type of limb-girdle muscular dystrophy (LGMD). The company, a spin-off from Genethon, a Paris-based genetics research laboratory, has submitted an application asking three European…
The Newborn Screening Pilot program run in New York by Parent Project Muscular Dystrophy (PMDD) is now complete, having screened more than 36,000 babies across the state for a muscular dystrophy over the last two years. Significantly high levels of creatine kinase, an enzyme released by damaged muscle cells that can indicate…
Sir Isaac Newton’s third law of motion states that for every action on an object there is an equal and opposite reaction. In 1686, his law was groundbreaking, but in 2021, we take its simplicity for granted. There is a consequence for every action taken. When a bullet discharges…
Flying can be an ordeal for many people with disabilities who rely on wheelchairs to get from point A to point B. Sometimes disabled travelers get to their destination only to realize their wheelchair or scooter is broken or missing. In 2019, the year after airlines were required to release…
On a Facebook page that documents our family’s journey with Duchenne muscular dystrophy, I recently posted about how invitations to birthday parties and friends’ houses are not typical for my three boys with Duchenne, especially with them using chairs full time. However, the post celebrated the fact that my…
The COVID-19 pandemic has had a substantial impact on the mental health and quality of life of people affected by muscular dystrophy (MD). One big underlying issue for the families of those with Duchenne and Becker muscular dystrophies is that the pandemic has caused many patients to…
Over the past few weeks, several people have asked me how I can be in a good mood nearly all the time when so many issues surround me. So, this week, I want to elaborate on my thought processes, and try to explain positive thinking, why I am the way…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to AOC 1001, an investigational treatment for myotonic dystrophy type 1, known as DM1, a late-developing muscle disorder. The designation will enable AOC 1001’s developer, Avidity Biosciences, to have more frequent interactions with the FDA throughout…
I think all moms need strong shoulders and extra arms. We carry purses, diaper bags, backpacks, and discarded jackets. Duchenne moms carry more, including sometimes our kids and their equipment, although I’ll admit that power wheelchairs are great for carrying bags without tipping over, unlike manual chairs and strollers.
New data across clinical trials of SRP-9001 show the investigative gene therapy induces sustained functional improvements in people with Duchenne muscular dystrophy (DMD), and does so with good tolerability, Sarepta Therapeutics, its developer, announced. The company, in presenting these data, also offered more details about the recently launched and pivotal…
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