In recent years scientists have learned that the protein utrophin can substitute for the dystrophin protein that is missing in muscular dystrophy. Researchers have now discovered that utrophin can reduce damage to energy-producing cell components known as mitochondria, whose deterioration is linked to MD. The reduction in damage improved the muscle…
The U.S. Food and Drug Administration has taken a step that will allow Sarepta Therapeutics to start clinical trials of its Duchenne muscular dystrophy therapy SRP-5051. That move was accepting the company’s Investigational New Drug application for SRP-5051. The therapy, known as a peptide phosphorodiamidate morpholino oligomer, is designed to…
Wave Life Sciences has started a Phase 1 clinical trial of a treatment for a gene malfunction linked to the most common form of Duchenne muscular dystrophy. Genes consists of dozens of components called exons. If an exon is missing, a gene can fail to produce a protein the body needs.
In this video from Gawker, Rich Juzwiak talks to comedian Steve Way about how having muscular dystrophy affects his life and how it impacts his comedy. MORE:Â Cyclists ride from Seattle to New York for facioscapulohumeral muscular dystrophy Way talks about the misconceptions people have…
These videos show how people living with muscular dystrophy (MD) can smash the stereotypes associated with having a disability, and lead rich and full lives. First up is 35-year-old Michael Klinkhamer, who has Becker muscular dystrophy. In this video shared by Story Hive, Michael explains that he spends all his…
The U.S. Food and Drug Administration (FDA) has cleared an investigational new drug application for a micro-dystrophin gene therapy program by Sarepta Therapeutics and Nationwide Children’s Hospital, in clinical development for Duchenne muscular dystrophy (DMD). A Phase 1/2a clinical trial is now enrolling participants and should begin dosing…
The Hope for Gus Foundation and its signature Everest to End Duchenne fundraising trek to Mount Everest Base Camp have raised $100,000 to fund new gene-editing research that could lead to new treatments for Duchenne muscular dystrophy (DMD). The nine-member U.S. Everest to End Duchenne team…
When you have a friend or family member who’s experiencing health problems, your first thought might be to pray for physical healing. At times, it feels as if that’s the only way to help. But, as someone living with a chronic illness/disability, physical healing may not be…
The U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for the GALGT2 gene therapy program for Duchenne muscular dystrophy (DMD), Sarepta Therapeutics and Nationwide Children’s Hospital recently announced. Following the FDA clearance, Nationwide Children’s will continue to advance the program by initiating a…
Managing a chronic illness can be difficult. There are many different medications to take (often at different times), appointments to remember, symptoms to keep track of, and lots of information to absorb. Thankfully, living in a digital age means that there are numerous mobile apps that can help you manage…
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