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 […]
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 […]
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 […]
Approach: Cardiac stem cell derived therapy
Funding Impact: Initiation of Phase 1/2 study (HOPE Clinical Trial)
Successful Exit: Stock Return
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 […]
My husband Paul and I have dreamed of this day since our son Hawken was diagnosed with Duchenne almost 15 years ago! We are overwhelmed with joy to announce […]
We are sharing the following update from BioMarin. We would like to update the patient community in light of the recent announcement regarding the EMA marketing application. BioMarin is […]
I imagine that by now you have heard the news that BioMarin has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) in Europe. Like you, I am heartbroken and […]
