Muscular dystrophy affects both adults and children all around the world. The degenerative muscle disease can impact both mobility and life expectancy, which is why so many committed organizations and people are working hard to find a cure for this debilitating disease. In today’s blog, we’ll highlight some recent news impacting those living with muscular dystrophy around the world. Learn more, below.
Eteplirsen fails to move forward in the U.S.
Many American parents and patients were hopeful that an experimental new drug called eteplirsen–which could purportedly help those living with Duchenne MD–would be given the greenlight during an FDA committee review meeting. Unfortunately, the panel of experts found little evidence of the drug’s efficiency in curing the disease, which will lessen its likelihood of being approved by the FDA. Many supporters had packed the hearing and spoke up to share their stories of the drug’s usefulness in slowing mobility loss, but the ultimate committee decision makes the fate of the drug unclear.
Ataluren receives the go-ahead in the UK
A different experimental drug called ataluren has recently received the greenlight from UK’s National Institute for Health and Care Excellence. The institute has published guidelines on the use of the drug to delay the need for a wheelchair in young boys with Duchenne MD. Though a deal has not yet been finalized with NHS England, this is an exciting advancement that could soon allow around 50 children to begin using the drug to hamper the progression of their disease.
While neither ataluren nor eteplirsen would act as a cure for muscular dystrophy, they could go a long way in improving outcomes for those living with the disease. Muscular dystrophy impacts tens of thousands of people around the globe and the loss of mobility is one of the most devastating impacts of this disease. To learn more about what you can do to help fight muscular dystrophy, visit the Muscular Dystrophy Association website and pledge your support!
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