Supporting Sarepta, Providing Hope

For those who battle against Duchenne muscular dystrophy, there is more hope now than ever. Recent medical breakthroughs in the form of effective drugs are slowing the disease’s progression and giving patients, families and supporters belief in a positive future.

Hope is more than a feeling, and here at CureDuchenne we take that belief and put it into action. Several years ago we became a catalyst for hope in action when we provided early funding to enable Sarepta (then AVI BioPharma) to move forward with the development of eteplirsen. Now eteplirsen is ready for a review by the FDA to possibly become available for about 13% of the boys with Duchenne.

Although, eteplirsen will not help my 19 year old son, Hawken, we are confident that the approval of this first drug will pave the way for faster approval for subsequent drugs and more interest and investment in Duchenne by biotech and pharmaceutical companies.

We believe the voices of the actual boys and parents, and their physicians, who have seen these benefits are crucially important. But, ultimately, the FDA will base its decision on scientific data, and we believe Sarepta has provided substantial data which proves the effectiveness of eteplirsen.  And we remain hopeful that the FDA will grant accelerated approval to eteplirsen.

CureDuchenne has brought Duchenne parents and key opinion leaders together with the FDA to discuss the importance of approving drugs that have shown to be effective in real-life.  We have sponsored cTAP, a collaboration between the pharmaceutical and biotech companies that are developing drugs for Duchenne, and created a statistical model to help them design more efficient trials and to help the FDA understand the natural history of Duchenne.

The Sarepta Advisory Committee meeting is April 25, just days away.  I look forward to speaking during the Open Public Hearing in support of eteplirsen along with other Duchenne families.  We are looking forward to hearing from patients and parents who have an important story to tell of their eteplirsen experience.

The Ad Comm will also no doubt be a reunion of sorts as hundreds of Duchenne parents and activists will meet in Washington DC for this occasion. If you are coming to Ad Comm, please email me (debra@cureduchenne.org). I would like to connect with you to share stories, and share hope. I look forward to seeing dear friends and meeting new Duchenne families.

We appreciate the entire Duchenne community supporting this review of eteplirsen. Together, we can cure Duchenne!