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 […]
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 […]
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 […]
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. […]
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 […]
The final decisions could be made today on the 21st Century Cures Act and the OPEN ACT – a provision that could be transformational for rare disease patients. CureDuchenne […]
Understanding a patient’s motivation for treatment is the key for a physical therapist to empower that patient for the best outcome. For a patient with Duchenne muscular dystrophy, a […]
Thank you to Marathon Pharmaceuticals for providing updated information about deflazacort access and regulatory status during a webinar this week. The webinar addressed Marathon’s Expanded Access Program for deflazacort and […]
As a parent of a son with Duchenne I have been frustrated that the data presented to the FDA does not match the patient experience. I personally know so […]
