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…
Discussion
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…
Discussion
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…
Discussion
I recently read a New York Times article about stress. Whether you live with a disability or not, stress is a universal phenomenon. Life is full of stressful moments both large and small that can overwhelm the best of us. In the article, the author asks three questions designed…
Discussion
I know what you’re thinking: another awareness day. One more health observance to briefly acknowledge for 24 hours, then mindlessly scroll past once it’s over. But for ultra-rare neuromuscular conditions, awareness is everything. Disease-specific awareness days actually matter. They create exposure, support, belonging, hope, and collaboration that can have a…
Becoming educated and informed about muscular dystrophy is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your muscular dystrophy journey.
There is no cure for muscular dystrophy yet, but here you can find more information about current therapeutic approaches, as well as experimental treatments that are in the pipeline.
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