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190 results

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Page 1
Financing drug development via adaptive platform trials.
Cho J, Sorets E, Chaudhuri S, De Mattos A, Drake K, Cudkowicz ME, Ortiz R, Hasenoehrl M, Chase M, Harkey B, Paganoni S, Frishkopf J, Lo AW. Cho J, et al. Among authors: cudkowicz me. PLoS One. 2025 Jul 2;20(7):e0325826. doi: 10.1371/journal.pone.0325826. eCollection 2025. PLoS One. 2025. PMID: 40601717 Free PMC article.
Opportunities for improving therapy development in ALS.
Bruijn L, Cudkowicz M; ALS Clinical Trials Working Group. Bruijn L, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Jun;15(3-4):169-73. doi: 10.3109/21678421.2013.872662. Epub 2014 Jan 29. Amyotroph Lateral Scler Frontotemporal Degener. 2014. PMID: 24472060
Plasma tau and phosphorylated tau at T181 are altered in amyotrophic lateral sclerosis.
Petrozziello T, Mizerak E, Krishnamoorthy A, Donahue RA, Castillo Torres AL, Monsanto RZB, Hammerschlag BL, Fillingham B, Kivisäkk P, Timmons J, Fox K, Arnold SE, Cohen J, Klee J, Paganoni S, Cudkowicz ME, Chibnik LB, Berry JD, Sadri-Vakili G. Petrozziello T, et al. Among authors: cudkowicz me. medRxiv [Preprint]. 2025 Jun 27:2025.06.26.25330363. doi: 10.1101/2025.06.26.25330363. medRxiv. 2025. PMID: 40666341 Free PMC article. Preprint.
Hospitalizations as an outcome measure in COURAGE-ALS.
Rudnicki SA, Al-Chalabi A, Andrews JA, Chio A, Corcia P, Couratier P, Cudkowicz ME, De Carvalho M, Genge A, Hardiman O, Heiman-Patterson T, Henderson RD, Ingre C, Johnston W, Ludolph A, Maragakis NJ, Miller TM, Mora JS, Petri S, Simmons Z, Van Den Berg LH, Zinman L, Herder KE, Kupfer S, Malik FI, Meng L, Simkins TJ, Wei J, Wolff AA, Shefner JM; Courage-Als study group. Rudnicki SA, et al. Among authors: cudkowicz me. Amyotroph Lateral Scler Frontotemporal Degener. 2025 Jun 12:1-10. doi: 10.1080/21678421.2025.2515907. Online ahead of print. Amyotroph Lateral Scler Frontotemporal Degener. 2025. PMID: 40503807
Operational Development and Launch of an Adaptive Platform Trial in Amyotrophic Lateral Sclerosis: Processes and Learnings From the First Four Regimens of the HEALEY ALS Platform Trial.
Harkey BA, Distefano S, Pagliaro JA, Heyd L, Chase M, Igne C, Yu H, Sherman AV, Dagostino D, Tustison E, Changkuon G, Hall M, Kittle G, Connolly MR, Giacomelli E, Scirocco E, Berry JD, Babu S, Shefner J, Macklin EA, Chibnik LB, De Mattos A, Drake K, Kamp C, McGarry A, Torti M, Small C, Bulat A, Cudkowicz ME, Paganoni S; HEALEY ALS Platform Trial Study Group. Harkey BA, et al. Among authors: cudkowicz me. Muscle Nerve. 2025 Aug;72(2):294-305. doi: 10.1002/mus.28442. Epub 2025 May 26. Muscle Nerve. 2025. PMID: 40420561
Reldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial.
Shefner JM, Cudkowicz ME, Genge A, Hardiman O, Al-Chalabi A, Andrews JA, Chio A, Corcia P, Couratier P, de Carvalho M, Heiman-Patterson T, Henderson RD, Ingre C, Johnston W, Ludolph A, Maragakis NJ, Miller TM, Mora 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; COURAGE-ALS Study Group. Shefner JM, et al. Among authors: cudkowicz me. JAMA Neurol. 2025 May 1;82(5):477-485. doi: 10.1001/jamaneurol.2025.0241. JAMA Neurol. 2025. PMID: 40126464 Free PMC article. Clinical Trial.
CNM-Au8 in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial.
Writing Committee for the HEALEY ALS Platform Trial; Berry JD, Maragakis NJ, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Stommel EW, Chase M, Pothier L, Harkey BA, Yu H, Sherman A, Shefner J, Hall M, Kittle G, Babu S, Andrews J, D'Agostino D, Tustison E, Scirocco E, Giacomelli E, Alameda G, Locatelli E, Ho D, Quick A, Ajroud-Driss S, Katz J, Heitzman D, Appel SH, Shroff S, Felice KJ, Simmons Z, Miller T, Olney N, Weiss MD, Goutman SA, Fernandes JA Jr, Jawdat O, Owegi MA, Foster L, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson C, Ladha S, Heiman-Patterson T, Caress J, Swenson A, Peltier A, Lewis R, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff CE, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Glass J, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Rynders A, Evan J, Evan J, Hartford A, Sepassi M, Ho KS, Glanzman R, Greenberg B, Hotchkin MT, Paganoni S, Cudkowicz ME; HEALEY ALS Platform Trial Study Group. Writing Committee for the HEALEY ALS Platform Trial, et al. Among authors: cudkowicz me. JAMA. 2025 Feb 17;333(13):1138-49. doi: 10.1001/jama.2024.27643. Online ahead of print. JAMA. 2025. PMID: 40067821
Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial.
Writing Committee for the HEALEY ALS Platform Trial; Shefner JM, Oskarsson B, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Heiman-Patterson T, Chase M, Pothier L, Harkey BA, Yu H, Sherman AV, Hall M, Kittle G, Berry JD, Babu S, Andrews J, D'Agostino D, Tustison E, Scirocco E, Giacomelli E, Alameda G, Locatelli E, Ho D, Quick A, Ajroud-Driss S, Katz J, Heitzman D, Appel SH, Shroff S, Felice K, Maragakis NJ, Simmons Z, Miller TM, Olney N, Weiss MD, Goutman SA, Fernandes JA, Jawdat O, Owegi MA, Foster LA, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson CE, Ladha S, Caress JB, Swenson A, Peltier A, Lewis RA, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff C, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Leitner ML, Chen K, Goldberg YP, Cohen Y, Geva M, Hayden MR, Paganoni S, Cudkowicz ME; HEALEY ALS Platform Trial Study Group. Writing Committee for the HEALEY ALS Platform Trial, et al. Among authors: cudkowicz me. JAMA. 2025 Feb 17;333(13):1128-37. doi: 10.1001/jama.2024.26429. Online ahead of print. JAMA. 2025. PMID: 40067755
Verdiperstat in Amyotrophic Lateral Sclerosis: Results From the Randomized HEALEY ALS Platform Trial.
Writing Committee for the HEALEY ALS Platform Trial; Andrews J, Paganoni S, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Young E, Chase M, Pothier L, Harkey B, Yu H, Sherman A, Shefner J, Hall M, Kittle G, Connolly MR, Berry JD, D'Agostino D, Tustison E, Giacomelli E, Scirocco E, Alameda G, Locatelli E, Ho D, Quick A, Heitzman D, Ajroud-Driss S, Appel SH, Shroff S, Katz J, Felice K, Maragakis NJ, Simmons Z, Goutman SA, Olney N, Miller T, Fernandes JA, Ilieva H, Jawdat O, Weiss MD, Foster L, Vu T, Ladha S, Owegi MA, Newman DS, Arcila-Londono X, Jackson CE, Swenson A, Heiman-Patterson T, Caress J, Fee D, Peltier A, Lewis R, Rosenfeld J, Walk D, Johnson K, Elliott M, Kasarskis EJ, Rutkove S, McIlduff CE, Bedlack R, Elman L, Goyal NA, Rezania K, Twydell P, Benatar M, Glass J, Cohen JA, Jones V, Zilliox L, Wymer JP, Beydoun SR, Shah J, Pattee GL, Martinez-Thompson J, Nayar S, Granit V, Donohue M, Grossman K, Campbell DJ, Qureshi IA, Cudkowicz ME, Babu S. Writing Committee for the HEALEY ALS Platform Trial, et al. Among authors: cudkowicz me. JAMA Neurol. 2025 Apr;82(4):333-343. doi: 10.1001/jamaneurol.2024.5249. Epub 2025 Feb 17. JAMA Neurol. 2025. PMID: 40067754 Free PMC article. Clinical Trial.
190 results