Rare Diseases

This sections highlights clinical protocols for a range of drugs.

Burosumab

HSE Burosumab (Crysvita) Protocol (PDF, 525 KB, 6 pages)

XLH001 HSE Burosumab Patient Eligibility Form (PDF, 261 KB, 1 page)

Voretigene Neparvovec

HSE Guidelines for the Treatment of Inherited Retinal Dystrophy with Vortigene Neparvovec (PDF, 1.1 MB, 7 pages)