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134 related items for PubMed ID: 38294974

  • 1. Trial of N-Acetyl-l-Leucine in Niemann-Pick Disease Type C.
    Bremova-Ertl T, Ramaswami U, Brands M, Foltan T, Gautschi M, Gissen P, Gowing F, Hahn A, Jones S, Kay R, Kolnikova M, Arash-Kaps L, Marquardt T, Mengel E, Park JH, Reichmannová S, Schneider SA, Sivananthan S, Walterfang M, Wibawa P, Strupp M, Martakis K.
    N Engl J Med; 2024 Feb 01; 390(5):421-431. PubMed ID: 38294974
    [Abstract] [Full Text] [Related]

  • 2. Efficacy and safety of N-acetyl-L-leucine in Niemann-Pick disease type C.
    Bremova-Ertl T, Claassen J, Foltan T, Gascon-Bayarri J, Gissen P, Hahn A, Hassan A, Hennig A, Jones SA, Kolnikova M, Martakis K, Raethjen J, Ramaswami U, Sharma R, Schneider SA.
    J Neurol; 2022 Mar 01; 269(3):1651-1662. PubMed ID: 34387740
    [Abstract] [Full Text] [Related]

  • 3. Efficacy and safety of N-acetyl-L-leucine in patients with ataxia telangiectasia: A randomized, double-blind, placebo-controlled, crossover clinical trial.
    Beyraghi-Tousi M, Sahebkar A, Houra M, Sarvghadi P, Jamialahmadi T, Bagheri R, Tavallaie S, Gumpricht E, Saberi-Karimian M.
    Eur J Paediatr Neurol; 2024 May 01; 50():57-63. PubMed ID: 38669738
    [Abstract] [Full Text] [Related]

  • 4. N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study.
    Fields T, M Bremova T, Billington I, Churchill GC, Evans W, Fields C, Galione A, Kay R, Mathieson T, Martakis K, Patterson M, Platt F, Factor M, Strupp M.
    Trials; 2023 May 29; 24(1):361. PubMed ID: 37248494
    [Abstract] [Full Text] [Related]

  • 5. Safety and Efficacy of Acetyl-DL-Leucine in Certain Types of Cerebellar Ataxia: The ALCAT Randomized Clinical Crossover Trial.
    Feil K, Adrion C, Boesch S, Doss S, Giordano I, Hengel H, Jacobi H, Klockgether T, Klopstock T, Nachbauer W, Schöls L, Steiner KM, Stendel C, Timmann D, Naumann I, Mansmann U, Strupp M, ALCAT Study Group.
    JAMA Netw Open; 2021 Dec 01; 4(12):e2135841. PubMed ID: 34905009
    [Abstract] [Full Text] [Related]

  • 6. Effects of acetyl-DL-leucine on cerebellar ataxia (ALCAT trial): study protocol for a multicenter, multinational, randomized, double-blind, placebo-controlled, crossover phase III trial.
    Feil K, Adrion C, Teufel J, Bösch S, Claassen J, Giordano I, Hengel H, Jacobi H, Klockgether T, Klopstock T, Nachbauer W, Schöls L, Stendel C, Uslar E, van de Warrenburg B, Berger I, Naumann I, Bayer O, Müller HH, Mansmann U, Strupp M.
    BMC Neurol; 2017 Jan 10; 17(1):7. PubMed ID: 28068987
    [Abstract] [Full Text] [Related]

  • 7. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment.
    Mengel E, Patterson MC, Da Riol RM, Del Toro M, Deodato F, Gautschi M, Grunewald S, Grønborg S, Harmatz P, Héron B, Maier EM, Roubertie A, Santra S, Tylki-Szymanska A, Day S, Andreasen AK, Geist MA, Havnsøe Torp Petersen N, Ingemann L, Hansen T, Blaettler T, Kirkegaard T, Í Dali C.
    J Inherit Metab Dis; 2021 Nov 10; 44(6):1463-1480. PubMed ID: 34418116
    [Abstract] [Full Text] [Related]

  • 8. Efficacy and Safety of N-Acetyl-l-Leucine in Children and Adults With GM2 Gangliosidoses.
    Martakis K, Claassen J, Gascon-Bayari J, Goldschagg N, Hahn A, Hassan A, Hennig A, Jones S, Kay R, Lau H, Perlman S, Sharma R, Schneider S, Bremova-Ertl T.
    Neurology; 2023 Mar 07; 100(10):e1072-e1083. PubMed ID: 36456200
    [Abstract] [Full Text] [Related]

  • 9. Acetyl-dl-leucine in Niemann-Pick type C: A case series.
    Bremova T, Malinová V, Amraoui Y, Mengel E, Reinke J, Kolníková M, Strupp M.
    Neurology; 2015 Oct 20; 85(16):1368-75. PubMed ID: 26400580
    [Abstract] [Full Text] [Related]

  • 10. Folic acid supplementation and malaria susceptibility and severity among people taking antifolate antimalarial drugs in endemic areas.
    Crider K, Williams J, Qi YP, Gutman J, Yeung L, Mai C, Finkelstain J, Mehta S, Pons-Duran C, Menéndez C, Moraleda C, Rogers L, Daniels K, Green P.
    Cochrane Database Syst Rev; 2022 Feb 01; 2(2022):. PubMed ID: 36321557
    [Abstract] [Full Text] [Related]

  • 11. The Effects of N-Acetyl-L-Leucine on the Improvement of Symptoms in a Patient with Multiple Sulfatase Deficiency.
    Saberi-Karimian M, Houra M, Jamialahmadi T, Sarvghadi P, Nikbaf M, Akhlaghi S, Sahebkar A.
    Cerebellum; 2023 Dec 01; 22(6):1250-1256. PubMed ID: 36482027
    [Abstract] [Full Text] [Related]

  • 12. Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial.
    Ory DS, Ottinger EA, Farhat NY, King KA, Jiang X, Weissfeld L, Berry-Kravis E, Davidson CD, Bianconi S, Keener LA, Rao R, Soldatos A, Sidhu R, Walters KA, Xu X, Thurm A, Solomon B, Pavan WJ, Machielse BN, Kao M, Silber SA, McKew JC, Brewer CC, Vite CH, Walkley SU, Austin CP, Porter FD.
    Lancet; 2017 Oct 14; 390(10104):1758-1768. PubMed ID: 28803710
    [Abstract] [Full Text] [Related]

  • 13. A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia.
    Fields T, Patterson M, Bremova-Ertl T, Belcher G, Billington I, Churchill GC, Davis W, Evans W, Flint S, Galione A, Granzer U, Greenfield J, Karl R, Kay R, Lewi D, Mathieson T, Meyer T, Pangonis D, Platt FM, Tsang L, Verburg C, Factor M, Strupp M.
    Trials; 2021 Jan 22; 22(1):84. PubMed ID: 33482890
    [Abstract] [Full Text] [Related]

  • 14. Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II trial.
    Sharma R, Hastings C, Staretz-Chacham O, Raiman J, Paucar M, Spiegel R, Murray B, Hurst B, Liu B, Kjems L, Hrynkow S.
    Mol Genet Metab Rep; 2023 Sep 22; 36():100988. PubMed ID: 37670901
    [Abstract] [Full Text] [Related]

  • 15. Safety and efficacy of riluzole in spinocerebellar ataxia type 2 in France (ATRIL): a multicentre, randomised, double-blind, placebo-controlled trial.
    Coarelli G, Heinzmann A, Ewenczyk C, Fischer C, Chupin M, Monin ML, Hurmic H, Calvas F, Calvas P, Goizet C, Thobois S, Anheim M, Nguyen K, Devos D, Verny C, Ricigliano VAG, Mangin JF, Brice A, Tezenas du Montcel S, Durr A.
    Lancet Neurol; 2022 Mar 22; 21(3):225-233. PubMed ID: 35063116
    [Abstract] [Full Text] [Related]

  • 16. Safety and Efficacy of Imatinib for Hospitalized Adults with COVID-19: A structured summary of a study protocol for a randomised controlled trial.
    Emadi A, Chua JV, Talwani R, Bentzen SM, Baddley J.
    Trials; 2020 Oct 28; 21(1):897. PubMed ID: 33115543
    [Abstract] [Full Text] [Related]

  • 17. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial.
    Writing Group, Edaravone (MCI-186) ALS 19 Study Group.
    Lancet Neurol; 2017 Jul 28; 16(7):505-512. PubMed ID: 28522181
    [Abstract] [Full Text] [Related]

  • 18. Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial.
    Hastings C, Liu B, Hurst B, Cox GF, Hrynkow S.
    Mol Genet Metab; 2022 Dec 28; 137(4):309-319. PubMed ID: 36279795
    [Abstract] [Full Text] [Related]

  • 19. Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials.
    Cortina-Borja M, Te Vruchte D, Mengel E, Amraoui Y, Imrie J, Jones SA, I Dali C, Fineran P, Kirkegaard T, Runz H, Lachmann R, Bremova-Ertl T, Strupp M, Platt FM.
    Orphanet J Rare Dis; 2018 Aug 16; 13(1):143. PubMed ID: 30115089
    [Abstract] [Full Text] [Related]

  • 20. Don't Fear the Reefer-Evidence Mounts for Plant-Based Cannabidiol as Treatment for Epilepsy.
    Perry MS.
    Epilepsy Curr; 2019 Aug 16; 19(2):93-95. PubMed ID: 30955420
    [Abstract] [Full Text] [Related]

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