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Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations (HST11)

Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies (confirmed biallelic RPE65 gene mutations)

Highly specialised technologies guidance Published October 2019

Patisiran for treating hereditary transthyretin amyloidosis (HST10)

Evidence-based recommendations on patisiran (Onpattro) for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy

Highly specialised technologies guidance Published August 2019

Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST12)

Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 (CLN2) in children

Highly specialised technologies guidance Published November 2019

Rapid tests for group A streptococcal infections in people with a sore throat (DG38)

Evidence-based recommendations on rapid tests for group A streptococcal (strep A) infections in people aged 5 and over with a sore throat

Diagnostics guidance Published November 2019

Therapeutic monitoring of TNF-alpha inhibitors in rheumatoid arthritis (DG36)

Evidence-based recommendations on enzyme-linked immunosorbent assay (ELISA) tests for therapeutic monitoring of TNF-alpha inhibitors in rheumatoid arthritis

Diagnostics guidance Published July 2019

Point-of-care creatinine devices to assess kidney function before CT imaging with intravenous contrast (DG37)

Evidence-based recommendations on point-of-care creatinine devices to assess kidney function before CT imaging with intravenous contrast

Diagnostics guidance Published November 2019