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
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
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