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 […]
EMFLAZA is the First Drug in the U.S. Approved for Use by Patients Age Five and Older with Duchenne Regardless of Genetic Mutation
Marathon Pharmaceuticals, LLC (Marathon), a U.S. research-based biopharmaceutical company focused solely on the development of new treatments for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted approval of EMFLAZA™ (deflazacort) for the treatment of Duchenne muscular dystrophy in patients 5 years and older. Duchenne, a severe form of muscular dystrophy, is a rare disease and fatal genetic disorder that affects about 15,000 people in the United States.1
The U.S. Food and Drug Administration today approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness. Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system.
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 […]
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced top-line safety and efficacy results for Part B of the MoveDMD® trial of edasalonexent (CAT-1004) for the treatment of Duchenne muscular dystrophy (DMD).
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. […]
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 […]
