‘Right to Try 2.0’ would bring innovative medicine to desperate Americans ...Middle East

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 four-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 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 c

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