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Drug Treatment for Lysosomal Storage Disorders (All ages), Commissioning Policy (CP55). January 2023 (PDF, 420Kb)
Sapropterin for treating hyperphenylalaninaemia (HPA) in phenylketonuria (PKU) and tetrahydrobiopterin (BH4) disorders, Policy Position (PP223), January 2023 (Saesneg) (PDF, 464Kb)
Volanesorsen for treating familial chylomicronaemia syndrome, Policy Position Statement, (PP217). October 2021 (Saesneg) (PDF, 285Kb)