Rare Disease Families Voice Concern Over Drug Pricing Policies
Rare diseases affect an estimated 25-30 million Americans, yet 95% lack FDA-approved treatments. Families affected by conditions like SLC6A1-related disorders and Anti-IgLON5 Autoimmune Encephalitis voice concerns about drug pricing policies that could slow medical innovation. Advocates urge lawmakers to protect research investment while sharing stories of diagnostic challenges and treatment breakthroughs.