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327 results

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Page 1
Effects of AFQ056 on language learning in fragile X syndrome.
Berry-Kravis E, Abbeduto L, Hagerman R, Coffey CS, Cudkowicz M, Erickson CA, McDuffie A, Hessl D, Ethridge L, Tassone F, Kaufmann WE, Friedmann K, Bullard L, Hoffmann A, Veenstra-VanderWeele J, Staley K, Klements D, Moshinsky M, Harkey B, Long J, Fedler J, Klingner E, Ecklund D, Costigan M, Huff T, Pearson B; NeuroNEXT FXLEARN Investigators. Berry-Kravis E, et al. Among authors: cudkowicz m. J Clin Invest. 2023 Aug 31;134(5):e171723. doi: 10.1172/JCI171723. J Clin Invest. 2023. PMID: 37651202 Free PMC article. Clinical Trial.
Negative effect of treatment with mGluR5 negative allosteric modulator AFQ056 on blood biomarkers in young individuals with Fragile X syndrome.
Protic D, Breeze E, Mendoza G, Zafarullah M, Abbeduto L, Hagerman R, Coffey C, Cudkowicz M, Durbin-Johnson B, Ashwood P, Berry-Kravis E, Erickson CA, Filipink R, Gropman A, Lehwald L, Maxwell-Horn A, Morris S, Bennett AP, Prock L, Talboy A, Tartaglia N, Veenstra-VanderWeele J, Tassone F. Protic D, et al. Among authors: cudkowicz m. SAGE Open Med. 2024 Sep 29;12:20503121241282401. doi: 10.1177/20503121241282401. eCollection 2024. SAGE Open Med. 2024. PMID: 39483619 Free PMC article.
Motor, cognitive, and functional declines contribute to a single progressive factor in early HD.
Schobel SA, Palermo G, Auinger P, Long JD, Ma S, Khwaja OS, Trundell D, Cudkowicz M, Hersch S, Sampaio C, Dorsey ER, Leavitt BR, Kieburtz KD, Sevigny JJ, Langbehn DR, Tabrizi SJ; TRACK-HD, COHORT, CARE-HD, and 2CARE Huntington Study Group Investigators. Schobel SA, et al. Among authors: cudkowicz m. Neurology. 2017 Dec 12;89(24):2495-2502. doi: 10.1212/WNL.0000000000004743. Epub 2017 Nov 15. Neurology. 2017. PMID: 29142089 Free PMC article.
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
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 May 26. doi: 10.1002/mus.28442. Online ahead of print. 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.
An Expanded Access Protocol of RNS60 in Amyotrophic Lateral Sclerosis.
Addy G, Scirocco E, Gelevski D, Rohrer M, Roderick A, McCormack M, Weiss Sadan A, Scalia J, Parikh N, Giacomelli E, Locatelli M, Neel DV, D'Agostino D, Leite A, Yu H, Sherman AV, Mock J, Kalmes A, Luppino S, Babu S, Berry J, Cudkowicz M, Paganoni S. Addy G, et al. Among authors: cudkowicz m. Muscle Nerve. 2025 Jul;72(1):124-129. doi: 10.1002/mus.28398. Epub 2025 Mar 21. Muscle Nerve. 2025. PMID: 40116017 Free PMC article. Clinical Trial.
327 results