FDA Approves Gene Therapy For Phase 1 Human Clinical Trials!


I'm beyond excited to share with you the BEST NEWS! The FDA has approved Gene Therapy for human clinical trials starting early next year. The trial will include newly diagnosed children 0-9 months old. If the trial is successful, the flood gates will open for more clinical trials and more children to be included and this could be the CURE for SMA.

Thank you Dr. Kaspar at Nationwide Children's Hospital and The Ohio State University for your tireless work and huge thank you to all of the SMA Foundations, Charities, families and friends that helped in any way to raise funds and awareness! We are so hopeful!