Here is a summary of today’s call with PTC’s Patient Advocacy and Commercial Teams Thank you PTC for hosting a call for advocacy leaders this morning to introduce the EMFLAZA team and give us a preview of the upcoming launch of this drug. PTC is committed to bringing treatments to rare diseases where there are […]
After her 10-month-old nephew was diagnosed with Duchenne muscular dystrophy, Karin Gorman was inspired to learn more about the disease and find a way to make a difference in the lives of families and individuals affected by Duchenne. A fatal genetic disease with no cure, Duchenne muscular dystrophy causes muscle deterioration and affects more than […]
If you told someone your child had cancer, they would immediately have a sense of the disease and potentially even a sense of the treatment options. People know about chemo, radiation and surgery. They don’t know much about rare diseases like Duchenne muscular dystrophy, and they are less likely to know that there is no […]
  Haydee Bustos has a passion for helping kids, and through her experience as a physical therapist, she discovered an even deeper desire to help kids with Duchenne muscular dystrophy. A fatal genetic disease, Duchenne muscular dystrophy causes muscle deterioration leaving patients, mostly boys, wheelchair bound by their mid-teens and claims their lives by their […]
  CureDuchenne is sharing an expanded and updated open letter to the Duchenne community from Marathon Pharmaceuticals. An Open Letter to the Duchenne Community Dear Members of the Duchenne Community: I want to thank you all for your support and encouragement over the last several years as Marathon worked to bring EMFLAZA™ (deflazacort) through the […]
  CureDuchenne is sharing an expanded and updated open letter to the Duchenne community from Marathon Pharmaceuticals. An Open Letter to the Duchenne Community  Dear Members of the Duchenne Community: I want to thank you all for your support and encouragement over the last several years as Marathon worked to bring EMFLAZA™ (deflazacort) through the […]
  We are delighted that the U.S. Food and Drug Administration (FDA) has approved EMFLAZA (deflazacort) for the treatment of Duchenne. This is wonderful news for the Duchenne community to have access to this drug in the U.S. We are grateful for Marathon Pharmaceuticals for conducting the multiple studies necessary for FDA approval. When your […]
  Today, the FDA approved the use of EMFLAZA (deflazacort) for Duchenne patients over the age of five years old.  It is routine for the FDA to designate specific patient populations on the “label” that are approved to receive a drug.  The reason the FDA excluded younger patients for EMFLAZA is because there has not […]

Move DMD Trial Update

Blog, Research
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  The MoveDMD trial with edasalonexent (CAT-1004) in Duchenne muscular dystrophy (DMD) has just completed Part B, and Catabasis has released the top-line results.   Summary: The top-line results that we are reporting are for Part B of a 3-part study in young boys with DMD. Positive results were reported for Part A in 2016. […]
  Calling all Texans.  Take action today and let your voices be heard.  Call your Texas State Senator and ask them to support Medicaid funding for the treatment of Duchenne. Overview The Texas Legislative Budget Board (LBB) is made up of a panel of state legislators who must approve all new Medicaid expenditures above $500,000 […]
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