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Asfotase alfa for treating paediatric-onset hypophosphatasia in children aged under 16 years, Policy Position Statement (PP156), November 2023 (Saesneg) (PDF, 263Kb)
Burosumab for Treating X-linked Hypophosphataemia in Children and Young People, Policy Position (PP177), May 2019 (Saesneg) (PDF, 557Kb)
Canakinumab for treating periodic fever syndromes: TRAPS, HIDS/MKD and FMF (ages 2 and older), Policy Position Statement, (PP228). January 2022 (Saesneg) (PDF, 324Kb)
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 for children aged under 16 years, Policy Position Statement (PP262), November 2023 (Saesneg) (PDF, 233Kb)
Givosiran for Treating Acute Hepatic Porphyria for People Aged 12 Years and Older, Policy Position Statement, (PP252), April 2023 (Saesneg) (PDF, 131Kb)
National Acute Porphyria, Service Specification (CP166). February 2025 (Saesneg) (PDF, 274Kb)
Pasireotide for Cushing's Disease, Policy Position (PP155), July 2019 (Saesneg) (PDF, 252Kb)
Treatment options for Transthyretin Amyloidosis in Adults, Policy Position Statement (PP187), September 2023 (Saesneg) (PDF, 307Kb)