Last weekend, I had the honor, along with Dr. Mike Kelly, CureDuchenne’s Chief Scientific Officer, and other TACT members, to review Duchenne research projects. CureDuchenne was able to look out for the best interest of Duchenne patients by providing a scientific, patient organization and parent perspective.
The projects that we reviewed were:
- FibroGen Inc, Clinical trial of FG-3019 in patients with Duchenne muscular dystrophy
- Naproxcinod for the treatment of Duchenne muscular dystrophy
- Audentes Therapeutics Inc: Systemically delivered AAV8 gene therapy for X-linked myotubular myopathy (XLMTM)
Treat-NMD will be reporting on these reviews early next year.
CureDuchenne is honored to be on the TACT TREAT–NMD Advisory Committee for Therapeutics. This multidisciplinary body provides the neuromuscular community (clinicians, researchers, patient advocacy groups and industry) with independent and objective guidance on advancing new therapies (whether novel or repurposed) for neuromuscular diseases.
The TACT review provides applicants with transparent and consistent guidance and advice, in an educational and directional context, on the readiness of drugs and/or therapeutic targets for trial. TACT advice will be helpful for preparing funding applications and investigational drug applications, while the publication of a non-confidential summary will ensure the community receives expert feedback on the progress of the application.
Throughout the year, I am pleased to represent the Duchenne community, and I consider this review committee to be one of the most meaningful and impactful gathering of experts who are truly working to bring drugs to our boys in the most efficient manner.