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.

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Title	Intervention	Indication	Therapeutic Area	Year	Actions
Givinostat for Duchenne muscular dystrophy	Givinostat	Duchenne muscular dystrophy (DMD)	Genetic Disorders , Musculoskeletal System	2023	View \| Download
Human C1-esterase inhibitor (subcutaneous injection) for preventing hereditary angioedema	Human C1 esterase inhibitor (Berinert)	Hereditary angioedema (HAE)	Genetic Disorders , Immunology	2023	View \| Download
Inclisiran for primary hypercholesterolaemia and mixed dyslipidaemia	Inclisiran (ALN-PCSsc; ALN-60212)	Heterozygous familial hypercholesterolaemia (HeFH) , Primary hypercholesterolaemia	Endocrine Nutritional and Metabolic Disorders , Genetic Disorders	2019	View \| Download
Ivacaftor for cystic fibrosis in infants	Ivacaftor (Kalydeco; VX-770)	Cystic fibrosis	Genetic Disorders , Respiratory System	2023	View \| Download
Kaftrio (Elexacaftor/tezacaftor/ivacaftor) and ivacaftor for treating cystic fibrosis in children (2 to 5 years) with at least one F508del mutation in the CFTR gene	Kaftrio (fixed dose combination: Elexacaftor; Tezacaftor; Ivacaftor)	Cystic fibrosis	Genetic Disorders , Respiratory System	2022	View \| Download
Lanadelumab for prevention of attacks in adults and adolescents with Type I and Type II hereditary angioedema	Lanadelumab (SHP643; TAK-743; Takhzyro)	Hereditary angioedema (HAE)	Genetic Disorders , Immunology	2017	View \| Download
Lenadogene Nolparvovec (GS-010) for Vision Loss from Leber's Hereditary Optic Neuropathy Due to Mutation of the ND4 Gene	Lenadogene nolparvovec (Lumevoq; GS-010)	Leber's hereditary optic neuropathy (LHON)	Genetic Disorders , Ophthalmology	2017	View \| Download
Leniolisib for previously untreated activated phosphoinositide 3-kinase delta syndrome	Leniolisib	Activated PI3K delta syndrome	Genetic Disorders	2022	View \| Download
Lenti‐D for adrenoleukodystrophy in boys	Elivaldogene tavalentivec (Lenti-D)	Adrenoleukodystrophy (ALD)	Genetic Disorders , Neurology	2017	View \| Download
Leriglitazone for X-linked andrenoleukodystrophy	Leriglitazone (MIN-102)	X-linked adrenoleukodystrophy (X-ALD)	Endocrine Nutritional and Metabolic Disorders , Genetic Disorders , Neurology	2023	View \| Download