Search All Tech Briefings


Search technology briefings produced by NIHR Innovation Observatory. These are short reports on new or repurposed medicines that we have already sent to our stakeholders. Only those technologies (medicines) that are within the scope of our stakeholders will have a technology briefing available.  Search our technology briefings by intervention (drug or device), specific indication (disease or condition), or general therapeutic area.

| 91 Results

Title
Intervention Indication Therapeutic Area Year Actions
VX-659/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 12 years and older Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor) , VX-659 (Bamocaftor-potassium) Cystic fibrosis Genetic Disorders , Respiratory System 2019 View  |  Download
VX-659/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation and one minimal function mutation in patients aged 12 years and older Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor) , VX-659 (Bamocaftor-potassium) Cystic fibrosis Genetic Disorders , Respiratory System 2019 View  |  Download
VX-445/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 12 years and older Elexacaftor (VX-445) , Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2019 View  |  Download
VX-445/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation and one minimal function mutation in patients aged 12 years and older Elexacaftor (VX-445) , Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2019 View  |  Download
Vonicog alfa for Prophylaxis of Haemorrhage in Von Willebrand disease Vonicog alfa (recombinant von Willebrand factor) von Willebrand disease Genetic Disorders , Haematology 2023 View  |  Download
Venglustat for treating type 3 Gaucher disease after 1 therapy Venglustat Gaucher disease Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2023 View  |  Download
Venglustat for treating late onset GM2 gangliosidosis, GM1 gangliosidosis and other ultra-rare disease Venglustat GM1 gangliosidosis , GM2 gangliosidosis Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2023 View  |  Download
Vanzacaftor / tezacaftor / deutivacaftor for the treatment of cystic fibrosis Deutivacaftor , Tezacaftor (VX-661) , Vanzacaftor Cystic fibrosis Genetic Disorders , Respiratory System 2023 View  |  Download
Vamorolone for treating Duchenne muscular dystrophy Vamorolone (VBP15) Duchenne muscular dystrophy (DMD) Genetic Disorders , Musculoskeletal System 2021 View  |  Download
Troriluzole for spinocerebellar ataxia Troriluzole Spinocerebellar ataxia Genetic Disorders , Neurology 2024 View  |  Download
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