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
Trofinetide for Rett syndrome	Trofinetide (NNZ-2566)	Rett syndrome	Genetic Disorders , Neurology	2021	View \| Download
Topical sirolimus for angiofibroma in tuberous sclerosis complex	Sirolimus (Rapamune; NPC-12G; Opsiria; DE-109)	Tuberous sclerosis	Genetic Disorders	2021	View \| Download
Topical rapamycin cream for treating facial angiofibroma	Topical rapamycin cream	Facial angiofibroma	Cardiovascular System , Dermatology , Genetic Disorders	2023	View \| Download
Tiratricol for treating monocarboxylate transporter 8 deficiency (Allan-Herndon-Dudley syndrome)	Tiratricol	Monocarboxylate transporter 8 deficiency (Allan-Herndon-Dudley syndrome)	Genetic Disorders , Neurology	2022	View \| Download
Tideglusib for congenital myotonic dystrophy	Tideglusib (AMO-02; Zentylor; Nypta; NP031112)	Myotonic dystrophy	Genetic Disorders , Musculoskeletal System , Neurology	2020	View \| Download
Tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 6 to 11 years	Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor)	Cystic fibrosis	Genetic Disorders , Respiratory System	2019	View \| Download
Tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation and one residual mutation in patients aged 6 to 11 years	Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor)	Cystic fibrosis	Genetic Disorders , Respiratory System	2019	View \| Download
Tezacaftor and ivacaftor for cystic fibrosis homozygous or heterozygous for F508del mutation in patients aged 12 years and older who discontinued treatment with Orkambi	Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor)	Cystic fibrosis	Genetic Disorders , Respiratory System	2019	View \| Download
Setmelanotide for obesity in Bardet-Biedl syndrome and Alstrom syndrome	Setmelanotide (RM-493)	Hyperphagia , Obesity	Endocrine Nutritional and Metabolic Disorders , Gastroenterology , Genetic Disorders	2021	View \| Download
Sebetralstat for treating hereditary angioedema	Sebetralstat (KVD-900)	Hereditary angioedema (HAE)	Genetic Disorders , Immunology	2023	View \| Download