Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

210 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study.
Howard JF Jr, Bresch S, Genge A, Hewamadduma C, Hinton J, Hussain Y, Juntas-Morales R, Kaminski HJ, Maniaol A, Mantegazza R, Masuda M, Sivakumar K, Śmiłowski M, Utsugisawa K, Vu T, Weiss MD, Zajda M, Boroojerdi B, Brock M, de la Borderie G, Duda PW, Lowcock R, Vanderkelen M, Leite MI; RAISE Study Team. Howard JF Jr, et al. Lancet Neurol. 2023 May;22(5):395-406. doi: 10.1016/S1474-4422(23)00080-7. Lancet Neurol. 2023. PMID: 37059508 Clinical Trial.
Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis.
Quintana M, Saville BR, Vestrucci M, Detry MA, Chibnik L, Shefner J, Berry JD, Chase M, Andrews J, Sherman AV, Yu H, Drake K, Cudkowicz M, Paganoni S, Macklin EA; HEALEY ALS Platform Trial Study Group. Quintana M, et al. Ann Neurol. 2023 Sep;94(3):547-560. doi: 10.1002/ana.26714. Epub 2023 Jun 22. Ann Neurol. 2023. PMID: 37245090
Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study.
Granit V, Benatar M, Kurtoglu M, Miljković MD, Chahin N, Sahagian G, Feinberg MH, Slansky A, Vu T, Jewell CM, Singer MS, Kalayoglu MV, Howard JF Jr, Mozaffar T; MG-001 Study Team. Granit V, et al. Among authors: mozaffar t. Lancet Neurol. 2023 Jul;22(7):578-590. doi: 10.1016/S1474-4422(23)00194-1. Lancet Neurol. 2023. PMID: 37353278 Clinical Trial.
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
Kishnani PS, Diaz-Manera J, Toscano A, Clemens PR, Ladha S, Berger KI, Kushlaf H, Straub V, Carvalho G, Mozaffar T, Roberts M, Attarian S, Chien YH, Choi YC, Day JW, Erdem-Ozdamar S, Illarioshkin S, Goker-Alpan O, Kostera-Pruszczyk A, van der Ploeg AT, An Haack K, Huynh-Ba O, Tammireddy S, Thibault N, Zhou T, Dimachkie MM, Schoser B; COMET Investigator Group. Kishnani PS, et al. Among authors: mozaffar t. JAMA Neurol. 2023 Jun 1;80(6):558-567. doi: 10.1001/jamaneurol.2023.0552. JAMA Neurol. 2023. PMID: 37036722 Free PMC article. Clinical Trial.
Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study.
Bril V, Drużdż A, Grosskreutz J, Habib AA, Mantegazza R, Sacconi S, Utsugisawa K, Vissing J, Vu T, Boehnlein M, Bozorg A, Gayfieva M, Greve B, Woltering F, Kaminski HJ; MG0003 study team. Bril V, et al. Lancet Neurol. 2023 May;22(5):383-394. doi: 10.1016/S1474-4422(23)00077-7. Lancet Neurol. 2023. PMID: 37059507 Clinical Trial.
Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial.
Machado PM, McDermott MP, Blaettler T, Sundgreen C, Amato AA, Ciafaloni E, Freimer M, Gibson SB, Jones SM, Levine TD, Lloyd TE, Mozaffar T, Shaibani AI, Wicklund M, Rosholm A, Carstensen TD, Bonefeld K, Jørgensen AN, Phonekeo K, Heim AJ, Herbelin L, Barohn RJ, Hanna MG, Dimachkie MM; Arimoclomol in IBM Investigator Team of the Neuromuscular Study Group. Machado PM, et al. Among authors: mozaffar t. Lancet Neurol. 2023 Oct;22(10):900-911. doi: 10.1016/S1474-4422(23)00275-2. Lancet Neurol. 2023. PMID: 37739573 Free article. Clinical Trial.
CAR T-Cell Therapy in Autoimmune Disease.
Howard JF Jr, Vu T, Mozaffar T. Howard JF Jr, et al. Among authors: mozaffar t. N Engl J Med. 2024 May 2;390(17):1629-1631. doi: 10.1056/NEJMc2403705. N Engl J Med. 2024. PMID: 38692299 No abstract available.
Debamestrocel multimodal effects on biomarker pathways in amyotrophic lateral sclerosis are linked to clinical outcomes.
Lindborg SR, Goyal NA, Katz J, Burford M, Li J, Kaspi H, Abramov N, Boulanger B, Berry JD, Nicholson K, Mozaffar T, Miller R, Jenkins L, Baloh RH, Lewis R, Staff NP, Owegi MA, Dagher B, Blondheim-Shraga NR, Gothelf Y, Levy YS, Kern R, Aricha R, Windebank AJ, Bowser R, Brown RH Jr, Cudkowicz ME. Lindborg SR, et al. Among authors: mozaffar t. Muscle Nerve. 2024 Jun;69(6):719-729. doi: 10.1002/mus.28093. Epub 2024 Apr 9. Muscle Nerve. 2024. PMID: 38593477 Clinical Trial.
272nd ENMC international workshop: 10 Years of progress - revision of the ENMC 2013 diagnostic criteria for inclusion body myositis and clinical trial readiness. 16-18 June 2023, Hoofddorp, The Netherlands.
Lilleker JB, Naddaf E, Saris CGJ, Schmidt J, de Visser M, Weihl CC; 272nd ENMC workshop participants. Lilleker JB, et al. Neuromuscul Disord. 2024 Apr;37:36-51. doi: 10.1016/j.nmd.2024.03.001. Epub 2024 Mar 7. Neuromuscul Disord. 2024. PMID: 38522330
210 results