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
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
Lonafarnib for Hutchinson-Gilford Progeria syndrome and progeroid laminopathies Lonafarnib (Sarasar; EBP994; SCH 66336; Zokinvy) Progeria Genetic Disorders 2020 View  |  Download
Lumacafor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 12 to 23 months Orkambi (fixed dose combination: Lumacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2019 View  |  Download
Lumacaftor and Ivacaftor (Orkambi) for Cystic Fibrosis Homozygous for the F508del CFTR mutation in patients aged 2-5 years old Orkambi (fixed dose combination: Lumacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2018 View  |  Download
Lumacaftor and Ivacaftor (Orkambi) for Cystic Fibrosis Homozygous for the F508del Mutation in Patients Aged 6‐11 Years Orkambi (fixed dose combination: Lumacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2018 View  |  Download
Luspatercept for adult patients with beta-thalassemia who require red blood cell transfusions Luspatercept (ACE-536; Reblozyl) Beta thalassemia Genetic Disorders , Haematology 2018 View  |  Download
Luspatercept for the treatment of adults with non-transfusion dependent beta-thalassaemia Luspatercept (ACE-536; Reblozyl) Beta thalassemia Genetic Disorders , Haematology 2020 View  |  Download
Maralixibat chloride for Progressive familial intrahepatic cholestasis Maralixibat chloride (SHP625) Progressive familial intrahepatic cholestasis (PFIC) Genetic Disorders , Hepatology 2023 View  |  Download
Maralixibat for cholestatic liver disease in patients with Alagille syndrome Maralixibat chloride (SHP625) Alagille syndrome Genetic Disorders 2021 View  |  Download
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