A note from Debra

FDA Approves EMFLAZA (deflazacort) … Our Experience With Deflazacort

Posted on February 10, 2017 4:03 pm

 

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 have access to this drug in the U.S. We are grateful for Marathon Pharmaceuticals for conducting the multiple studies necessary for FDA approval.

When your child is diagnosed with a genetic disease that has no cure, you will consider every treatment option available to improve the quality of his or her life. This is exactly how we felt when our son Hawken was diagnosed with Duchenne muscular dystrophy nearly 15 years ago. We looked at everything, weighing risks and potential rewards as well as availability. We were happy to find deflazacort, a steroid that had been yielding positive results for boys with Duchenne.

We were hopeful when we began Hawken’s treatment with deflazacort on his sixth birthday. He had been exhibiting symptoms of Duchenne for some time, and we were very worried. Much to our delight, Hawken responded well and quickly. After a week on deflazacort, he ran across the playground. He began to have such better command of his legs that when I picked him he wrapped them around me! This had never happened before. My heart was so full of joy to experience such a quintessential parent moment. His energy and strength continued to increase. There were some side effects and we kept a close eye on his diet and that helped to mitigate those effects. Overall, the benefits far outweighed the concerns.

Hawken still takes deflazacort, and soon this drug will be readily available to every boy who battles Duchenne.  This is such an exciting step forward in treating this disease. This will be the first approved drug treatment for every Duchenne patient regardless of mutation. We appreciate Marathon’s commitment to helping patients have access to EMFLAZA. They have created comprehensive patient assistance program called EMFLAZACares.  For more information, visit www.EMFLAZA.comor call 1-844-EMFLAZA (1-844-363-5292).  Click here for FAQs.

The progress forward in treating and managing Duchenne is gaining momentum every day, and we are so proud to see this transformation. We are hopeful that before long a cure will be in our reach.

 

Additional Information about EMFLAZA (deflazacort) for Duchenne Patients under the Age of Five

Posted on 1:49 am

  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 Read more »

Move DMD Trial Update

Posted on January 31, 201710:58 pm

  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. Read more »