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.

| 61 Results

Title
Intervention Indication Therapeutic Area Year Actions
Odevixibat for cholestatic liver disease in Alagille syndrome patients Odevixibat (A4250) Alagille syndrome Genetic Disorders 2022 View  |  Download
Oleogel-S10 for Inherited Epidermolysis Bullosa Oleogel-S10 (Episalvan) Inherited epidermolysis bullosa (EB) Dermatology , Genetic Disorders 2018 View  |  Download
OTL-103 for Wiskott-Aldrich syndrome OTL-103 (GSK-2696275) Wiskott-Aldrich syndrome (WAS) Genetic Disorders , Immunology 2020 View  |  Download
OTL-200 for Metachromatic Leukodystrophy OTL-200 (GSK-2696274) Metachromatic leukodystrophy (MLD) Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2019 View  |  Download
Pegunigalsidase alfa for Fabry disease – first-line PRX-102 (Pegunigalsidase alfa) Fabry disease Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2020 View  |  Download
PXT3003 for Charcot-Marie-Tooth Disease Type 1A PXT-3003 (fixed dose combination: baclofen; Naltrexone hydrochloride; sorbitol) Charcot-Marie-Tooth disease Genetic Disorders , Neurology 2018 View  |  Download
rAAVrh74.MHCK7.micro-dystrophin for Duchenne muscular dystrophy Microdystrophin (SRP-9001; rAAVrh74.MHCK7) Duchenne muscular dystrophy (DMD) Genetic Disorders , Musculoskeletal System 2020 View  |  Download
Risdiplam for paediatric and adult patients with spinal muscular atrophy (SMA) Risdiplam (RG-7916; RO-7034067; Evrysdi) Spinal muscular atrophy (SMA) Genetic Disorders , Musculoskeletal System 2019 View  |  Download
Sarizotan for Respiratory Symptoms Associated Rett Syndrome Sarizotan (EMD-128130) Rett syndrome Genetic Disorders , Neurology 2018 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
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