Updating results

930 results

Sort: Relevance | Date

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

Proposed [GID-HST10022] 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

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] 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

OTL-200 for treating metachromatic leukodystrophy [ID1666]

Proposed [GID-HST10028] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Velmanase alfa for treating alpha-mannosidosis [ID800]

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

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

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

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

In development [GID-HST10026] Expected publication date: 08 September 2020

Highly specialised technologies guidance In development

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]

In development [GID-HST10015] Expected publication date: 03 June 2020

Highly specialised technologies guidance In development

Elosulfase alfa for treating mucopolysaccharidosis type IVa (review of HST2) [ID1643]

In development [GID-HST10027] Expected publication date: 11 November 2020

Highly specialised technologies guidance In development

Human alpha1-proteinase inhibitor for treating emphysema ID856

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

Highly specialised technologies guidance In development

Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

Evidence-based recommendations on culizumab (Soliris) for treating atypical haemolytic uraemic syndrome (aHUS)

Highly specialised technologies guidance Published January 2015

Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (HST3)

Evidence-based recommendations on ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene..

Highly specialised technologies guidance Published July 2016

Elosulfase alfa for treating mucopolysaccharidosis type IVa (HST2)

Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type IVa (MPS IVA)

Highly specialised technologies guidance Published December 2015

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

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

Burosumab for treating X-linked hypophosphataemia in children and young people (HST8)

Evidence-based recommendations on burosumab (Crysvita) for treating X-linked hypophosphataemia (XLH) in children and young people

Highly specialised technologies guidance Published October 2018

Eliglustat for treating type 1 Gaucher disease (HST5)

Evidence-based recommendations on eliglustat (Cerdelga) for treating type 1 Gaucher disease in adults

Highly specialised technologies guidance Published June 2017

Asfotase alfa for treating paediatric-onset hypophosphatasia (HST6)

Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children

Highly specialised technologies guidance Published August 2017

Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency (HST7)

Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency (ADA–SCID)

Highly specialised technologies guidance Published February 2018

Migalastat for treating Fabry disease (HST4)

Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16

Highly specialised technologies guidance Published February 2017

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

Inotersen for treating hereditary transthyretin amyloidosis (HST9)

Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis

Highly specialised technologies guidance Published May 2019

Filgotinib for treating moderate to severe rheumatoid arthritis ID1632

Proposed [GID-TA10541] Expected publication date: TBC

Technology appraisal guidance Proposed

Netarsudil for treating open angle glaucoma or ocular hypertension [ID1078]

Proposed [GID-TA10431] Expected publication date: TBC

Technology appraisal guidance Proposed

Luspatercept for treating beta-thalassaemia ID1554

Proposed [GID-TA10506] Expected publication date: 16 September 2020

Technology appraisal guidance Proposed

Solriamfetol for treating excessive waketime sleepiness caused by narcolepsy [ID1602]

Proposed [GID-TA10524] Expected publication date: 16 September 2020

Technology appraisal guidance Proposed

Eflornithine with sulindac for treating familial adenomatous polyposis ID1543

Proposed [GID-TA10536] Expected publication date: TBC

Technology appraisal guidance Proposed

Satralizumab for preventing relapses in neuromyelitis optica spectrum disorders [ID1319]

Proposed [GID-TA10544] Expected publication date: TBC

Technology appraisal guidance Proposed

Emapalumab for treating primary haemophagocytic lymphohistiocytosis (ID1438)

Proposed [GID-TA10527] Expected publication date: TBC

Technology appraisal guidance Proposed

Nintedanib for treating progressive fibrosing interstitial lung disease [ID1599]

Proposed [GID-TA10520] Expected publication date: TBC

Technology appraisal guidance Proposed

Lenadogene nolparvovec for treating Leber's hereditary optic neuropathy [ID1410]

Proposed [GID-TA10391] Expected publication date: TBC

Technology appraisal guidance Proposed

Upadacitinib for treating moderate to severe rheumatoid arthritis [ID1400]

Proposed [GID-TA10389] Expected publication date: 18 March 2020

Technology appraisal guidance Proposed

Fostamatinib for treating persistent or chronic immune thrombocytopenia [ID1087]

Proposed [GID-TA10387] Expected publication date: TBC

Technology appraisal guidance Proposed

Pitolisant hydrochloride for treating obstructive sleep apnoea ID1065

Proposed [GID-TA10385] Expected publication date: TBC

Technology appraisal guidance Proposed

Autologous haematopoietic stem cell transplantation for treating multiple sclerosis (ID1111)

Proposed [GID-TA10306] Expected publication date: TBC

Technology appraisal guidance Proposed

Rituximab for maintenance treatment of anti-neutrophil cytoplasmic antibody-associated vasculitis [ID1320]

Proposed [GID-TA10332] Expected publication date: TBC

Technology appraisal guidance Proposed

Avatrombopag for treating thrombocytopenia in people with chronic liver disease needing elective surgery (ID1105)

Proposed [GID-TA10348] Expected publication date: TBC

Technology appraisal guidance Proposed

Tocilizumab for treating systemic sclerosis (ID1396)

Proposed [GID-TA10346] Expected publication date: TBC

Technology appraisal guidance Proposed

Pegvaliase for treating phenylketonuria [ID1110]

Proposed [GID-TA10345] Expected publication date: TBC

Technology appraisal guidance Proposed

Brentuximab vedotin for untreated CD30-positive peripheral T-cell lymphoma ID1586

Proposed [GID-TA10514] Expected publication date: 12 August 2020

Technology appraisal guidance Proposed

Cladribine tablets for treating relapsing–remitting multiple sclerosis [ID2703]

Proposed [GID-TA10548] Expected publication date: 19 December 2019

Technology appraisal guidance Proposed

Treosulfan with fludarabine for non-malignant disease before allogeneic stem cell transplant [ID1540]

Proposed [GID-TA10453] Expected publication date: TBC

Technology appraisal guidance Proposed

Dulaglutide for treating type 2 diabetes [ID1451]

Proposed [GID-TA10439] Expected publication date: TBC

Technology appraisal guidance Proposed

Fingolimod for treating relapsing multiple sclerosis in children and young people [ID1378]

Proposed [GID-TA10336] Expected publication date: TBC

Technology appraisal guidance Proposed

Intravenous zanamivir for treating influenza in hospital [ID1196]

Proposed [GID-TA10298] Expected publication date: TBC

Technology appraisal guidance Proposed

Lusutrombopag for treating thrombocytopenia in people with chronic liver disease needing elective surgery [ID1149]

Proposed [GID-TA10297] Expected publication date: TBC

Technology appraisal guidance Proposed