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Page 1
Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease.
Lessard S, Rimmelé P, Ling H, Moran K, Vieira B, Lin YD, Rajani GM, Hong V, Reik A, Boismenu R, Hsu B, Chen M, Cockroft BM, Uchida N, Tisdale J, Alavi A, Krishnamurti L, Abedi M, Galeon I, Reiner D, Wang L, Ramezi A, Rendo P, Walters MC, Levasseur D, Peters R, Harris T, Hicks A. Lessard S, et al. Among authors: cockroft bm. Sci Rep. 2024 Oct 16;14(1):24298. doi: 10.1038/s41598-024-74716-7. Sci Rep. 2024. PMID: 39414860 Free PMC article. Clinical Trial.
Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study.
Leavitt AD, Konkle BA, Stine KC, Visweshwar N, Harrington TJ, Giermasz A, Arkin S, Fang A, Plonski F, Yver A, Ganne F, Agathon D, Resa MLA, Tseng LJ, Di Russo G, Cockroft BM, Cao L, Rupon J. Leavitt AD, et al. Among authors: cockroft bm. Blood. 2024 Feb 29;143(9):796-806. doi: 10.1182/blood.2022018971. Blood. 2024. PMID: 37871576 Free PMC article. Clinical Trial.
COURAGE-ALS: a randomized, double-blind phase 3 study designed to improve participant experience and increase the probability of success.
Shefner JM, Al-Chalabi A, Andrews JA, Chio A, De Carvalho M, Cockroft BM, Corcia P, Couratier P, Cudkowicz ME, Genge A, Hardiman O, Heiman-Patterson T, Henderson RD, Ingre C, Jackson CE, Johnston W, Lechtzin N, Ludolph A, Maragakis NJ, Miller TM, Mora Pardina JS, Petri S, Simmons Z, Van Den Berg LH, Zinman L, Kupfer S, Malik FI, Meng L, Simkins TJ, Wei J, Wolff AA, Rudnicki SA. Shefner JM, et al. Among authors: cockroft bm. Amyotroph Lateral Scler Frontotemporal Degener. 2023 Aug;24(5-6):523-534. doi: 10.1080/21678421.2023.2216223. Epub 2023 May 30. Amyotroph Lateral Scler Frontotemporal Degener. 2023. PMID: 37254449 Clinical Trial.
Correction to: Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study.
Rudnicki SA, Andrews JA, Duong T, Cockroft BM, Malik FI, Meng L, Wei J, Wolff AA, Genge A, Johnson NE, Tesi-Rocha C, Connolly AM, Darras BT, Felice K, Finkel RS, Shieh PB, Mah JK, Statland J, Campbell C, Habib AA, Kuntz NL, Oskoui M, Day JW. Rudnicki SA, et al. Among authors: cockroft bm. Neurotherapeutics. 2021 Jul;18(3):2130. doi: 10.1007/s13311-021-01120-8. Neurotherapeutics. 2021. PMID: 34731415 Free PMC article. No abstract available.
Prescription and acceptance of durable medical equipment in FORTITUDE-ALS, a study of reldesemtiv in ALS: post hoc analyses of a randomized, double-blind, placebo-controlled clinical trial.
Rudnicki SA, Andrews JA, Genge A, Jackson C, Lechtzin N, Miller TM, Cockroft BM, Malik FI, Meng L, Wei J, Wolff AA, Shefner JM; FORTITUDE-ALS STUDY GROUP. Rudnicki SA, et al. Among authors: cockroft bm. Amyotroph Lateral Scler Frontotemporal Degener. 2022 May;23(3-4):263-270. doi: 10.1080/21678421.2021.1946083. Epub 2021 Jul 5. Amyotroph Lateral Scler Frontotemporal Degener. 2022. PMID: 34218726 Clinical Trial.
Noninvasive ventilation use by patients enrolled in VITALITY-ALS.
Rudnicki SA, Andrews JA, Bian A, Cockroft BM, Cudkowicz ME, Hardiman O, Malik FI, Meng L, Wolff AA, Shefner JM; VITALITY-ALS STUDY GROUP. Rudnicki SA, et al. Among authors: cockroft bm. Amyotroph Lateral Scler Frontotemporal Degener. 2021 Nov;22(7-8):486-494. doi: 10.1080/21678421.2021.1904993. Epub 2021 Apr 1. Amyotroph Lateral Scler Frontotemporal Degener. 2021. PMID: 33792451 Clinical Trial.
Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study.
Rudnicki SA, Andrews JA, Duong T, Cockroft BM, Malik FI, Meng L, Wei J, Wolff AA, Genge A, Johnson NE, Tesi-Rocha C, Connolly AM, Darras BT, Felice K, Finkel RS, Shieh PB, Mah JK, Statland J, Campbell C, Habib AA, Kuntz NL, Oskoui M, Day JW. Rudnicki SA, et al. Among authors: cockroft bm. Neurotherapeutics. 2021 Apr;18(2):1127-1136. doi: 10.1007/s13311-020-01004-3. Epub 2021 Feb 23. Neurotherapeutics. 2021. PMID: 33624184 Free PMC article. Clinical Trial.
A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS.
Shefner JM, Andrews JA, Genge A, Jackson C, Lechtzin N, Miller TM, Cockroft BM, Meng L, Wei J, Wolff AA, Malik FI, Bodkin C, Brooks BR, Caress J, Dionne A, Fee D, Goutman SA, Goyal NA, Hardiman O, Hayat G, Heiman-Patterson T, Heitzman D, Henderson RD, Johnston W, Karam C, Kiernan MC, Kolb SJ, Korngut L, Ladha S, Matte G, Mora JS, Needham M, Oskarsson B, Pattee GL, Pioro EP, Pulley M, Quan D, Rezania K, Schellenberg KL, Schultz D, Shoesmith C, Simmons Z, Statland J, Sultan S, Swenson A, Berg LHVD, Vu T, Vucic S, Weiss M, Whyte-Rayson A, Wymer J, Zinman L, Rudnicki SA. Shefner JM, et al. Among authors: cockroft bm. Amyotroph Lateral Scler Frontotemporal Degener. 2021 May;22(3-4):287-299. doi: 10.1080/21678421.2020.1822410. Epub 2020 Sep 24. Amyotroph Lateral Scler Frontotemporal Degener. 2021. PMID: 32969758 Free PMC article. Clinical Trial.
A phase III trial of tirasemtiv as a potential treatment for amyotrophic lateral sclerosis.
Shefner JM, Cudkowicz ME, Hardiman O, Cockcroft BM, Lee JH, Malik FI, Meng L, Rudnicki SA, Wolff AA, Andrews JA; VITALITY-ALS Study Group. Shefner JM, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2019;0(0):1-11. doi: 10.1080/21678421.2019.1612922. Amyotroph Lateral Scler Frontotemporal Degener. 2019. PMID: 31081694 Clinical Trial.