‘Right to Try 2.0’ Would Bring Innovative Medicine to Desperate Americans

Five-year-old Olivia Riley of Arizona suffers from an extremely rare and fatal genetic brain disorder called metachromatic leukodystrophy (MLD). She is tragically in hospice care, unlikely to survive her childhood.

That her younger sister Keira, who has the very same disease, is a thriving 4-year-old is a testament to the power of an innovative gene therapy that saved Keira’s life as an infant, before she started showing symptoms.

Had the Riley family waited for the Food and Drug Administration (FDA) to approve that personalized treatment, Lenmeldy (OTL-200), which finally happened last week, it would have been too late for Keira. The Rileys had to move to Italy in 2020 so that Keira could get the care she needed.

Read the rest of the op-ed at The Hill. 

Naomi Lopez is a Senior Fellow at the Goldwater Institute.