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.

| 61 Results

Title
Intervention Indication Therapeutic Area Year Actions
Cannabidiol (Epidiolex) for Tuberous Sclerosis Complex - add-on therapy Cannabidiol (BRCX-014; Epidiolex; GW42003; GWP42003; GWP-42003; PG-DN-20WS) Tuberous sclerosis Genetic Disorders 2017 View  |  Download
CER-001 for Familial Primary Hypoalphalipoproteinemia CER-001 Familial primary hypoalphalipoproteinemia Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2018 View  |  Download
CTX001 for transfusion-dependent beta-thalassaemia CTX001 Beta thalassemia Genetic Disorders , Haematology 2021 View  |  Download
Cysteamine bitartrate (Procysbi) delayed release for Huntington’s Disease Cysteamine bitartrate (Procysbi; RP103; Lynovex; NM001; Cystadrops; Mercaptamine) Huntington's disease Genetic Disorders , Neurology 2017 View  |  Download
Cysteamine bitartrate for cystic fibrosis exacerbations Cysteamine bitartrate (Procysbi; RP103; Lynovex; NM001; Cystadrops; Mercaptamine) Cystic fibrosis Genetic Disorders , Respiratory System 2018 View  |  Download
Domagrozumab (PF-6252616) for Duchenne muscular dystrophy in children and adolescents Domagrozumab (PF-06252616) Duchenne muscular dystrophy (DMD) Genetic Disorders , Musculoskeletal System 2017 View  |  Download
Edasalonexent for Duchene muscular dystrophy Edasalonexent (CAT-1004) Duchenne muscular dystrophy (DMD) Genetic Disorders , Musculoskeletal System 2020 View  |  Download
Eflornithine in combination with Sulindac for Familial Adenomatus Polyposis Eflornithine (CPP-1X; Difluoromethylornithine; DFMO; Vaniqa; eflornithine hydrochloride monohydrate) , Sulindac Familial adenomatous polyposis (FAP) Gastroenterology , Genetic Disorders 2018 View  |  Download
Elexacaftor/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation and one minimal function mutation in patients aged 6 to 11 years Elexacaftor (VX-445) , Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2019 View  |  Download
Elexacaftor/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation in patients aged 12 years old and older Elexacaftor (VX-445) , Ivacaftor (Kalydeco; VX-770) , Tezacaftor (VX-661) Cystic fibrosis Genetic Disorders , Respiratory System 2020 View  |  Download
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