Search results

629 results

Sorted by Relevance . Sort by Date

  1. Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (HST3)

    Evidence-based recommendations on ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people aged

  2. Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

    Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children

  3. Givosiran for treating acute hepatic porphyria (HST16)

    Evidence-based recommendations on givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 and over

  4. Odevixibat for treating progressive familial intrahepatic cholestasis (HST17)

    Evidence-based recommendations on odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6 months and older

  5. Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)

    Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children

  6. Inotersen for treating hereditary transthyretin amyloidosis (HST9)

    Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis

  7. Burosumab for treating X-linked hypophosphataemia in children and young people (HST8)

    Evidence-based recommendations on burosumab (Crysvita) for treating X-linked hypophosphataemia (XLH) in children and young people

  8. Asfotase alfa for treating paediatric-onset hypophosphatasia (HST6)

    Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children

  9. Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST12)

    Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children

  10. Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations (HST11)

    Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherit

  11. Patisiran for treating hereditary transthyretin amyloidosis (HST10)

    Evidence-based recommendations on patisiran (Onpattro) for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy

  12. Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over (HST20)

    Evidence-based recommendations on selumetinib (Koselugo) for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over

  13. Belimumab for treating active autoantibody-positive systemic lupus erythematosus (TA752)

    Evidence-based recommendations on belimumab (Benlysta) for treating active autoantibody-positive systemic lupus erythematosus

  14. Cenobamate for treating focal onset seizures in epilepsy (TA753)

    Evidence-based recommendations on cenobamate (Ontozry) for treating focal onset seizures with or without secondary generalised seizures in adults with drug

  15. Mogamulizumab for previously treated mycosis fungoides and Sézary syndrome (TA754)

    Evidence-based recommendations on mogamulizumab (Poteligeo) for previously treated mycosis fungoides and Sézary syndrome in

  16. Selpercatinib for previously treated RET fusion-positive advanced non-small-cell lung cancer (TA760)

    Evidence-based recommendations on selpercatinib (Retsevmo) for previously treated RET fusion-positive advanced non-small-cell lung cancer in adults

  17. Solriamfetol for treating excessive daytime sleepiness caused by narcolepsy (TA758)

    Evidence-based recommendations on solriamfetol (Sunosi) for treating excessive daytime sleepiness caused by narcolepsy in adults

  18. Apalutamide with androgen deprivation therapy for treating high-risk hormone-relapsed non-metastatic prostate cancer (TA740)

    Evidence-based recommendations on apalutamide with androgen deprivation therapy for treating high-risk hormone-relapsed non-metastatic prostate cancer in adults

  19. Apalutamide with androgen deprivation therapy for treating hormone-sensitive metastatic prostate cancer (TA741)

    Evidence-based recommendations on apalutamide with androgen deprivation therapy for treating hormone-sensitive metastatic prostate cancer in adults

  20. Dupilumab for treating severe asthma with type 2 inflammation (TA751)

    Evidence-based recommendations on dupilumab (Dupixent) for treating severe asthma with type 2 inflammation that is inadequately controlled in people 12 years an

  21. Eribulin for treating locally advanced or metastatic breast cancer after 1 chemotherapy regimen (TA515)

    Evidence-based recommendations on eribulin (Halaven) for treating locally advanced or metastatic (secondary) breast cancer in adults who have had only 1 course

  22. Ixazomib with lenalidomide and dexamethasone for treating relapsed or refractory multiple myeloma (TA505)

    Evidence-based recommendations on ixazomib (Ninlaro), with lenalidomide and dexamethasone for relapsed or refractory multiple myeloma in adults

  23. Ribociclib with an aromatase inhibitor for previously untreated, hormone receptor-positive, HER2-negative, locally advanced or metastatic breast cancer (TA496)

    Evidence-based recommendations on ribociclib (Kisqali) as initial endocrine-based therapy for hormone-receptor positive, human epidermal growth factor receptor

  24. Abemaciclib with an aromatase inhibitor for previously untreated, hormone receptor-positive, HER2-negative, locally advanced or metastatic breast cancer (TA563)

    Evidence-based recommendations on abemaciclib (Verzenios) for treating locally advanced or metastatic, hormone receptor-positive, human epidermal growth factor

  25. Encorafenib with binimetinib for unresectable or metastatic BRAF V600 mutation-positive melanoma (TA562)

    Evidence-based recommendations on encorafenib (Braftovi) with binimetinib (Mektovi) for treating unresectable or metastatic BRAF V600 mutation-positive melanoma

  26. Venetoclax with rituximab for previously treated chronic lymphocytic leukaemia (TA561)

    Evidence-based recommendations on venetoclax (Venclyxto) with rituximab for previously treated chronic lymphocytic leukaemia in adults

  27. Liposomal cytarabine–daunorubicin for untreated acute myeloid leukaemia (TA552)

    Evidence-based recommendations on liposomal cytarabine–daunorubicin (Vyxeos) for untreated acute myeloid leukaemia (AML) in adults

  28. Gemtuzumab ozogamicin for untreated acute myeloid leukaemia (TA545)

    Evidence-based recommendations on emtuzumab oxogamicin (Mylotarg) for untreated acute myeloid leukaemia (AML) in people aged 15 years and over

  29. Dabrafenib with trametinib for adjuvant treatment of resected BRAF V600 mutation-positive melanoma (TA544)

    Evidence-based recommendations on dabrafenib (Tafinlar) with trametinib (Mekinist) for resected stage III, BRAF V600 mutation-positive melanoma in adults

  30. Tofacitinib for treating active psoriatic arthritis after inadequate response to DMARDs (TA543)

    Evidence-based recommendations on tofacitinib (Xeljanz) for treating active psoriatic arthritis after inadequate response to DMARDs in adults

  31. Inotuzumab ozogamicin for treating relapsed or refractory B-cell acute lymphoblastic leukaemia (TA541)

    Evidence-based recommendations on inotuzumab ozogamicin (Besponsa) for relapsed/refractory CD22-positive B-cell precursor acute lymphoblastic leukaemia (ALL)

  32. Ixekizumab for treating active psoriatic arthritis after inadequate response to DMARDs (TA537)

    Evidence-based recommendations on ixekizumab (Taltz) for treating active psoriatic arthritis after inadequate response to DMARDs in adults

  33. Palbociclib with an aromatase inhibitor for previously untreated, hormone receptor-positive, HER2-negative, locally advanced or metastatic breast cancer (TA495)

    Evidence-based recommendations on palbociclib (Ibrance) as initial endocrine-based therapy for hormone receptor-positive, HER2-negative, locally advanced or

  34. Nivolumab for treating relapsed or refractory classical Hodgkin lymphoma (TA462)

    Evidence-based recommendations on nivolumab (Opdivo) for treating relapsed or refractory classical Hodgkin lymphoma in adults