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114 results

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Page 1
Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis.
Neel DV, Baselga-Garriga C, Benson M, Keegan M, Chase M, D'Agostino D, Drake K, Hagar JL, Hasenoehrl MG, Kulesa-Kelley J, Leite A, Mohapatra S, Portaro SM, Pothier LM, Rosenthal J, Sherman AV, Yu H, McCaffrey A, Ho D, Luppino S, Bedlack R, Heitzman D, Ajroud-Driss S, Katz J, Felice K, Whitaker C, Ladha S, Alameda G, Locatelli E, Qureshi IA, Hotchkin MT, Hayden MR, Cudkowicz ME, Babu S, Berry JD, Paganoni S. Neel DV, et al. Among authors: felice k. Muscle Nerve. 2024 Aug;70(2):232-239. doi: 10.1002/mus.28169. Epub 2024 Jun 6. Muscle Nerve. 2024. PMID: 38842106
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.
A randomized, placebo-controlled, double-blind phase IIb trial evaluating the safety and efficacy of tirasemtiv in patients with amyotrophic lateral sclerosis.
Shefner JM, Wolff AA, Meng L, Bian A, Lee J, Barragan D, Andrews JA; BENEFIT-ALS Study Group. Shefner JM, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2016 Jul-Aug;17(5-6):426-435. doi: 10.3109/21678421.2016.1148169. Epub 2016 Mar 16. Amyotroph Lateral Scler Frontotemporal Degener. 2016. PMID: 26982815 Clinical Trial.
A Double-Blinded, Randomized, Placebo-Controlled Trial to Evaluate Efficacy, Safety, and Tolerability of Single Doses of Tirasemtiv in Patients with Acetylcholine Receptor-Binding Antibody-Positive Myasthenia Gravis.
Sanders DB, Rosenfeld J, Dimachkie MM, Meng L, Malik FI; Tirasemtiv in Myasthenia Gravis Study Group. Sanders DB, et al. Neurotherapeutics. 2015 Apr;12(2):455-60. doi: 10.1007/s13311-015-0345-y. Neurotherapeutics. 2015. PMID: 25742919 Free PMC article. Clinical Trial.
Correction to: A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot-Marie-Tooth type 1A.
Attarian S, Young P, Brannagan TH, Adams D, Van Damme P, Thomas FP, Casanovas C, Kafaie J, Tard C, Walter MC, Péréon Y, Walk D, Stino A, de Visser M, Verhamme C, Amato A, Carter G, Magy L, Statland JM, Felice K. Attarian S, et al. Among authors: felice k. Orphanet J Rare Dis. 2024 Apr 1;19(1):142. doi: 10.1186/s13023-024-03110-3. Orphanet J Rare Dis. 2024. PMID: 38561848 Free PMC article. No abstract available.
Protein-extending ACTN2 frameshift variants cause variable myopathy phenotypes by protein aggregation.
Ranta-Aho J, Felice KJ, Jonson PH, Sarparanta J, Yvorel C, Harzallah I, Touraine R, Pais L, Austin-Tse CA, Ganesh VS, O'Leary MC, Rehm HL, Hehir MK, Subramony S, Wu Q, Udd B, Savarese M. Ranta-Aho J, et al. Among authors: felice kj. Ann Clin Transl Neurol. 2024 Sep;11(9):2392-2405. doi: 10.1002/acn3.52154. Epub 2024 Aug 2. Ann Clin Transl Neurol. 2024. PMID: 39095936 Free PMC article.
Rare ACTN2 Frameshift Variants Resulting in Protein Extension Cause Distal Myopathy and Hypertrophic Cardiomyopathy through Protein Aggregation.
Ranta-Aho J, Felice KJ, Jonson PH, Sarparanta J, Palmio J, Tasca G, Sabatelli M, Yvorel C, Harzallah I, Touraine R, Pais L, Austin-Tse CA, Ganesh V, O'Leary MC, Rehm HL, Hehir MK, Subramony S, Wu Q, Udd B, Savarese M. Ranta-Aho J, et al. Among authors: felice kj. medRxiv [Preprint]. 2024 Jan 17:2024.01.17.23298671. doi: 10.1101/2024.01.17.23298671. medRxiv. 2024. PMID: 38293186 Free PMC article. Preprint.
114 results