My son is dying - families like mine can’t afford FDA delays and red tape

The mother of a 14-year-old with Duchenne muscular dystrophy has invited FDA Commissioner Dr. Martin Makary to visit her home, arguing that the agency's slow approval process for rare disease treatments is costing children their lives.

Her son Ryu has been in a wheelchair for years. She's spent over four decades fighting the same terminal illness that killed her brothers. The emotional and financial toll is staggering — full-time caregiving, specialized equipment, and the constant fear that the next treatment breakthrough will come too late.

Her argument is not that the FDA should abandon safety standards, but that the current process creates an impossible calculus for families facing terminal diagnoses. While the agency reviews data, conducts trials, and processes applications, children with progressive diseases lose ground they never regain. A drug that works at age 10 may not work at age 14 after the disease has progressed further.

The tension between safety and speed is real. The FDA's accelerated approval pathway exists partly in response to this problem, but rare diseases present unique challenges: small patient populations make traditional clinical trials difficult, and the financial incentives for developing treatments for conditions affecting fewer than 200,000 Americans are thin.

Commissioner Makary, who has spoken about streamlining the agency, now faces pressure from families who want words turned into action. The invitation to see Ryu's daily reality is a challenge: come see what your processes look like from the living room of a family running out of time.

**What This Means For You:** Rare diseases affect 30 million Americans. If you or someone you know is affected, advocacy matters — the FDA responds to public pressure and congressional oversight. Support organizations pushing for faster rare disease approvals, and if you're eligible for clinical trials, consider participating. Your data could be the difference between a treatment existing and not existing.