Enzyme Replacement Therapy

Sebelipase

HSE Prescribing Protocol for Sebelipase alfa for the Treatment of Lysosomal Acid Lipase Deficiency (PDF, size 961.7 KB, 14 pages)

Fabry Disease

HSE Guidelines for the Treatment of Fabry Disease (PDF, 1.13 MB, 17 pages)

Cerliponase

HSE Guidelines for the Treatment of Battens Disease with Cerliponase (PDF, size 1.1 MB, 10 pages)

Sapropterin

HSE Prescribing Guideline for Sapropterin Dihydrochloride (Kuvan®) Treatment (PDF, size 897.2 KB, 11 pages)

Late-Onset Pompe Disease

HSE Prescribing Protocol for Alglusidase alfa (Myozyme) for Late-Onset Pompe Disease (PDF, size 773 KB, 5 pages)

MPS IVa

HSE Prescribing Protocol for Elosulfase alfa (Vimizim) for Mucopolysaccharidosis Type IVa (PDF, size 741 KB, 6 pages)