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
  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 […]
  Darcy Peach discovered her calling to become a physical therapist while undergoing treatment for a volleyball injury in high school. During her treatment sessions, Darcy’s physical therapist took a great deal of care and time in treating her.  She witnessed first-hand the therapeutic and relationship building benefits that occur during the process of physical […]
  Marathon submitted New Drug Applications (NDAs) to the Food and Drug Administration (FDA) last June for deflazacort. The NDAs have been accepted for filing and review. The FDA expects to complete its review of the applications in early February. If approved, deflazacort will become accessible in the U.S. and it would be the second […]
CureDuchenne has always been focused on finding and funding treatments to help EVERYONE affected with Duchenne, and to TREAT THE WHOLE DISEASE.  We are very proud of our comprehensive portfolio of research projects and we continue to be the research leader for the Duchenne community. Over the past five years we have pioneered duplication mutation […]


CureDuchenne is pleased to share the following updates from Pfizer pertaining to the on-going multicenter Phase II clinical trial of the investigational compound, domagrozumab (PF-06252616) in boys with Duchenne muscular dystrophy (DMD). Domagrozumab is an experimental, infused, anti-myostatin monoclonal antibody.  Myostatin is a naturally occurring protein in muscles that helps control muscle growth; it is […]


Guest blog by Paul Miller Our son Hawken was diagnosed with Duchenne in the fall of 2002.  Debra and I rolled up our sleeves and began a journey we had not anticipated.   We found there were many variables to managing this disease.  One of these is physical therapy.  Maintaining the health of the muscles is […]
As a community, we need to continue our efforts to find a cure for every single person impacted by Duchenne. That means funding research that treats all aspects of the disease.  That includes anti-inflammatory and anti-fibrosis therapies, gene therapy and gene editing; and cardiac therapies that address the leading cause of death for Duchenne.  As […]
Yesterday the House of Representatives passed the draft of the 21st Century Cures Act. It is slated for a vote in the Senate early next week.       The new draft includes: $4.8 billion in new funding for the National Institutes of Health (NIH) $500 million in new funding for the Food and Drug […]
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