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