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

Clear All Filters

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
Edasalonexent for Duchene muscular dystrophy	Edasalonexent (CAT-1004)	Duchenne muscular dystrophy (DMD)	Genetic Disorders , Musculoskeletal System	2020	View \| Download