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.

| 90 Results

Title
Intervention Indication Therapeutic Area Year Actions
Biotin for Adrenomyeloneuropathy without cerebral involvement Biotin (MD1003; Qizenday; Vitamin B7) Adrenomyeloneuropathy (AMN) Genetic Disorders , Neurology 2017 View  |  Download
Bis-choline tetrathiomolybdate for Wilson's disease Bis-choline tetrathiomolybdate (Decuprate; ATN-224; WTX101; ALXN1840) Wilson's disease Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2021 View  |  Download
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
Cipaglucosidase alfa/miglustat for treating children and adolescents with late-onset Pompe disease Cipaglucosidase alfa/miglustat (AT-GAA;;Zavesca,;OGT 918;BBDF101) Pompe disease Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2023 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 
Donidalorsen for the prophylactic treatment of hereditary angioedema Donidalorsen (ISIS 721744;IONIS-PKK-LRx) Hereditary angioedema (HAE) Genetic Disorders , Immunology 2024 View  |  Download
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