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Nationwide Children’s Hospital
Approach: Exon skipping (Duplication exon 2 mutations)
Funding Impact: Advanced pre-clinical IND package enabling clinical trial for Duchenne boys
An Open Letter to the Duchenne Community
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: […]
An Open Letter to the Duchenne Community
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: […]
FDA Approves EMFLAZA (deflazacort) … Our Experience With Deflazacort
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 […]
Additional Information about EMFLAZA (deflazacort) for Duchenne Patients under the Age of Five
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 […]
CureDuchenne Applauds the FDA Approval of Marathon’s EMFLAZA (deflazacort) for the Treatment of Duchenne Muscular Dystrophy
EMFLAZA is the First Drug in the U.S. Approved for Use by Patients Age Five and Older with Duchenne Regardless of Genetic Mutation
FDA Approves EMFLAZA™ (deflazacort) Tablets and Oral Suspension for the Treatment of Duchenne Muscular Dystrophy in Patients 5 Years and Older
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
FDA approves drug to treat Duchenne muscular dystrophy
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.
Move DMD Trial Update
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 […]