LHF Blog
Expert Physcian Testimonies on SRP-9001
Posted on May 8, 2023
Hear directly from the SRP-9001 PI's expert views on treatment effect in their patients.
Expert Physician Testimonies on SRP-9001
Later this week, the FDA will hold an Advisory Committee meeting to review the first-ever gene therapy to treat Duchenne Muscular Dystrophy. SRP-9001 (delandistrogene moxeparvovec) is among the most exciting developments in the history of Duchenne. Families who participated in the clinical trials have shared with the community how the treatment is changing the trajectory of the disease for their boys. Many of them will have the opportunity to speak during the open public forum portion of the Advisory Committee meeting and I look forward to hearing their testimonies.
Unfortunately, many of the physicians who led the studies for SRP-9001 will not have the same opportunity. The FDA used a lottery system to select speakers for the open public forum portion of the meeting and none of the study physicians who applied to speak were chosen. Given that Advisory Committee meetings of this type are focused on reviewing the data generated through the clinical trials, the voice of these specific investigators with open label experience in those trials is undeniably relevant to the discussion and arguably the most important due to their experience with the drug and treating the disease.
Since the physicians were not selected to speak at the Advisory Committee meeting, they recorded video testimonies and submitted them for the official record. Their experiences in the studies and what they’ve seen in their patients gives me even more confidence that gene therapy is going to make a huge difference for our community.
You can watch each of the video testimonies at the following links:
- Crystal Proud, Children's Hospital of the King's Daughters
- Emma Ciafaloni, University of Rochester
- Craig Zaidman, Washington University
- Perry Shieh, University of California, Los Angeles
I am hopeful that these video testimonies are taken into consideration as the committee reviews the wealth of evidence supporting this important new therapy for our boys with Duchenne.
Kelly Maynard, Little Hercules Foundation
Mother to Jackson, 16
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