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
Maralixibat for Progressive Familial Intrahepatic Cholestasis Type 2 Maralixibat chloride (SHP625) Progressive familial intrahepatic cholestasis (PFIC) Genetic Disorders , Hepatology 2020 View  |  Download
Mavorixafor for WHIM syndrome Mavorixafor (X4P-001) WHIM syndrome Genetic Disorders , Immunology 2021 View  |  Download
Mexiletine hydrochloride for treating myotonia in children and adolescents with myotonic disorders Mexiletine hydrochloride (Namuscla) Myotonia Genetic Disorders 2023 View  |  Download
Migalastat for Fabry disease in children aged 12 to 15 years Migalastat hydrochloride (Galafold; AT1001) Fabry disease Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2019 View  |  Download
Nitisinone for Alkaptonuria Nitisinone (Orfadin; SYN118) Alkaptonuria Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2019 View  |  Download
NPJ5008 for treating malignant hyperthermia NPJ5008 Malignant hyperthermia Genetic Disorders 2022 View  |  Download
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
Olezarsen for treating familial chylomicronaemia syndrome Olezarsen (ISIS 678354; AKCEA-APOCIII-LRx) Familial chylomicronaemia syndrome (FCS) Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2024 View  |  Download
Omaveloxolone for the treatment of Friedreich's ataxia Omaveloxolone Friedreich's ataxia Genetic Disorders , Neurology 2023 View  |  Download
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