Step-by-Step: Compassionate Use, Trial Aid & Back-to-School Flu Plan
A mother's story of navigating compassionate-use access to an orphan drug, building a back-to-school flu plan for her child with a rare disease, and finding travel and financial aid for trial participation.
Robert Maxwell
Sep 10, 2025
orphan drug access
gene therapy trials
lysosomal storage disorders
compassionate use requests
