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Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations ID1054

In development [GID-HST10025] Expected publication date: 09 October 2019

Highly specialised technologies guidance In development

Patient group directions (MPG2)

This guideline covers good practice for developing, authorising, using and updating patient group directions. It also offers advice on deciding whether a patient group direction is needed.

Medicines practice guideline Published August 2013 Last updated March 2017

Controlled drugs: safe use and management (NG46)

This guideline covers systems and processes for using and managing controlled drugs safely in all NHS settings except care homes. It aims to improve working practices to comply with legislation and have robust governance arrangements. It also aims to reduce the safety risks associated with controlled drugs.

NICE guideline Published April 2016

Developing and updating local formularies (MPG1)

This guideline covers good practice for developing and updating local formularies in line with statutory requirements. It supports developing formularies that reflect local needs, reduce variation in prescribing, and allow rapid adoption of new medicines and treatments.

Medicines practice guideline Published March 2014 Last updated October 2015

Managing medicines for adults receiving social care in the community (NG67)

This guideline covers medicines support for adults (aged 18 and over) who are receiving social care in the community. It aims to ensure that people who receive social care are supported to take and look after their medicines effectively and safely at home. It gives advice on assessing if people need help with managing their medicines, who should provide medicines support and how health and social care staff should work together.

NICE guideline Published March 2017

Antimicrobial stewardship: systems and processes for effective antimicrobial medicine use (NG15)

This guideline covers the effective use of antimicrobials (including antibiotics) in children, young people and adults. It aims to change prescribing practice to help slow the emergence of antimicrobial resistance and ensure that antimicrobials remain an effective treatment for infection.

NICE guideline Published August 2015

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Human alpha1-proteinase inhibitor for treating emphysema ID856

In development [GID-HST10017] Expected publication date: TBC

Highly specialised technologies guidance In development

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]

In development [GID-HST10015] Expected publication date: TBC

Highly specialised technologies guidance In development

OTL-101 for treating adenosine deaminase deficiency–severe combined immunodeficiency [ID1152]

Proposed [GID-HST10022] Expected publication date: TBC

Highly specialised technologies guidance Proposed

VTS-270 for treating Niemann-Pick type C1 (ID1267)

Proposed [GID-HST10020] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Ravulizumab for treating paroxysmal nocturnal haemoglobinuria ID1457

Proposed [GID-HST10023] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Onasemnogene abeparvovec for treating spinal muscular atrophy type 1 [ID1473]

In development [GID-HST10026] Expected publication date: 03 June 2020

Highly specialised technologies guidance In development

Afamelanotide for treating erythropoietic protoporphyria [ID927]

In development [GID-HST10009] Expected publication date: TBC

Highly specialised technologies guidance In development

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] Expected publication date: TBC

Highly specialised technologies guidance In development

Metreleptin for treating lipodystrophy (ID861)

In development [GID-HST10011] Expected publication date: TBC

Highly specialised technologies guidance In development

Lysosomal acid lipase deficiency - sebelipase alfa [ID737]

In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] Expected publication date: TBC

Highly specialised technologies guidance In development

Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 [ID943]

In development [GID-HST10008] Expected publication date: 27 November 2019

Highly specialised technologies guidance In development

Eteplirsen for treating Duchenne muscular dystrophy [ID1003]

In development [GID-HST10007] Expected publication date: TBC

Highly specialised technologies guidance In development

Drisapersen for the first-line treatment of Duchenne's muscular dystrophy [ID911]

In development [GID-HST10004] Expected publication date: TBC

Highly specialised technologies guidance In development