El Paso Mother Advocates for Faster FDA Approval of Rare Disease Treatments

Angelina Olivera, a resident of El Paso, expresses her frustration with the FDA's delays in approving treatments for rare diseases, particularly Duchenne muscular dystrophy, which affects her son Ryu. As Dr. Vinay Prasad departs from the FDA, Olivera highlights the urgent need for accelerated drug approvals, especially after the agency halted the only approved gene therapy for Duchenne following tragic patient deaths. Families of children with rare diseases are suffering as they wait for essential treatments that could prolong their lives. Olivera's advocacy at a Senate hearing has garnered bipartisan support, but she urges new FDA leadership to prioritize these urgent cases to prevent further decline in patients' health.

Key Details: • Ryu, 14, suffers from Duchenne muscular dystrophy, a condition with a shortened life expectancy. • FDA's recent decisions have led to increased delays and rejections of drug applications for rare diseases. • Senator Ron Johnson has initiated an investigation into the FDA's handling of treatments for children with rare diseases. • New FDA leadership is urged to expedite the approval process for critical therapies.