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.

| 87 Results

Title
Intervention Indication Therapeutic Area Year Actions
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
Elexacaftor/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 6 to 11 years old 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 and ivacaftor for treating cystic fibrosis without an F508del mutation and with an ELX/TEZ/IVA-responsive mutation in the CFTR gene in patients aged 6 years and older Ivacaftor (Kalydeco; VX-770) , Kaftrio (fixed dose combination: Elexacaftor; Tezacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2023 View  |  Download
Emapalumab for Primary Haemophagocytic Lymphohistiocytosis Emapalumab (NI-0501; Gamifant) Primary haemophagocytic lymphohistiocytosis (HLH) Genetic Disorders , Immunology 2018 View  |  Download
Eplontersen for hereditary transthyretin-mediated amyloid polyneuropathy Eplontersen (ION-682884;AKCEA TTR-LRx) Transthyretin-mediated amyloid polyneuropathy Genetic Disorders 2023 View  |  Download
Evinacumab for treating homozygous familial hypercholesterolemia in children Evinacumab (REGN1500) Homozygous familial hypercholesterolemia Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2022 View  |  Download
Fordadistrogene movaparvovec for Duchenne muscular dystrophy Fordadistrogene movaparvovec Duchenne muscular dystrophy (DMD) Genetic Disorders , Musculoskeletal System 2022 View  |  Download
Garadacimab for preventing attacks of hereditary angioedema Garadacimab Hereditary angioedema (HAE) Genetic Disorders , Immunology 2023 View  |  Download
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