Treatment for HT-1 (hereditary tyrosinemia) involves using the FDA-approved medication nitisinone. It is a synthetic, reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase, an enzyme producing harmful compounds connected to HT-1. In addition to taking Nitisinone, patients must follow a strict diet restricting their intake of tyrosine and phenylalanine. Report any unexpected eye problems, rashes, jaundice, or substantial bleeding immediately. With a daily dose cap of 2 mg/kg, the dosage…

A Managed Access Program (MAP) is a healthcare initiative designed to provide access to investigational or unapproved medications for patients with serious or life-threatening conditions. MAPs are developed in partnership with healthcare providers, regulatory bodies, and pharmaceutical companies. These programs address the urgent medical requirements of people who have exhausted all therapeutic…