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Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial.
Benatar M, Hansen T, Rom D, Geist MA, Blaettler T, Camu W, Kuzma-Kozakiewicz M, van den Berg LH, Morales RJ, Chio A, Andersen PM, Pradat PF, Lange D, Van Damme P, Mora G, Grudniak M, Elliott M, Petri S, Olney N, Ladha S, Goyal NA, Meyer T, Hanna MG, Quinn C, Genge A, Zinman L, Jabari D, Shoesmith C, Ludolph AC, Neuwirth C, Nations S, Shefner JM, Turner MR, Wuu J, Bennett R, Dang H, Sundgreen C; ORARIALS-01 trial team. Benatar M, et al. Among authors: petri s. Lancet Neurol. 2024 May 20:S1474-4422(24)00134-0. doi: 10.1016/S1474-4422(24)00134-0. Online ahead of print. Lancet Neurol. 2024. PMID: 38782015
Targeting low levels of MIF expression as a potential therapeutic strategy for ALS.
Alfahel L, Gschwendtberger T, Kozareva V, Dumas L, Gibbs R, Kertser A, Baruch K, Zaccai S, Kahn J, Thau-Habermann N, Eggenschwiler R, Sterneckert J, Hermann A, Sundararaman N, Vaibhav V, Van Eyk JE, Rafuse VF, Fraenkel E, Cantz T, Petri S, Israelson A. Alfahel L, et al. Among authors: petri s. Cell Rep Med. 2024 May 21;5(5):101546. doi: 10.1016/j.xcrm.2024.101546. Epub 2024 May 3. Cell Rep Med. 2024. PMID: 38703766 Free article.
Small peptide CSF fingerprint of amyotrophic lateral sclerosis.
Lumi R, Petri S, Siwy J, Latosinska A, Raad J, Zürbig P, Skripuletz T, Mischak H, Beige J. Lumi R, et al. Among authors: petri s. PLoS One. 2024 Apr 30;19(4):e0302280. doi: 10.1371/journal.pone.0302280. eCollection 2024. PLoS One. 2024. PMID: 38687737 Free PMC article.
Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy: A Nonrandomized Controlled Trial.
Schwartz O, Vill K, Pfaffenlehner M, Behrens M, Weiß C, Johannsen J, Friese J, Hahn A, Ziegler A, Illsinger S, Smitka M, von Moers A, Kölbel H, Schreiber G, Kaiser N, Wilichowski E, Flotats-Bastardas M, Husain RA, Baumann M, Köhler C, Trollmann R, Schwerin-Nagel A, Eisenkölbl A, Schimmel M, Fleger M, Kauffmann B, Wiegand G, Baumgartner M, Rauscher C, Cirak S, Gläser D, Bernert G, Hagenacker T, Goldbach S, Probst-Schendzielorz K, Lochmüller H, Müller-Felber W, Schara-Schmidt U, Walter MC, Kirschner J, Pechmann A; SMARTCARE study group. Schwartz O, et al. JAMA Pediatr. 2024 Apr 8:e240492. doi: 10.1001/jamapediatrics.2024.0492. Online ahead of print. JAMA Pediatr. 2024. PMID: 38587854
European Academy of Neurology (EAN) guideline on the management of amyotrophic lateral sclerosis in collaboration with European Reference Network for Neuromuscular Diseases (ERN EURO-NMD).
Van Damme P, Al-Chalabi A, Andersen PM, Chiò A, Couratier P, De Carvalho M, Hardiman O, Kuźma-Kozakiewicz M, Ludolph A, McDermott CJ, Mora JS, Petri S, Probyn K, Reviers E, Salachas F, Silani V, Tysnes OB, van den Berg LH, Villanueva G, Weber M. Van Damme P, et al. Among authors: petri s. Eur J Neurol. 2024 Jun;31(6):e16264. doi: 10.1111/ene.16264. Epub 2024 Mar 12. Eur J Neurol. 2024. PMID: 38470068
5qSMA: standardised retrospective natural history assessment in 268 patients with four copies of SMN2.
Vill K, Tacke M, König A, Baumann M, Baumgartner M, Steinbach M, Bernert G, Blaschek A, Deschauer M, Flotats-Bastardas M, Friese J, Goldbach S, Gross M, Günther R, Hahn A, Hagenacker T, Hauser E, Horber V, Illsinger S, Johannsen J, Kamm C, Koch JC, Koelbel H, Koehler C, Kolzter K, Lochmüller H, Ludolph A, Mensch A, Meyer Zu Hoerste G, Mueller M, Mueller-Felber W, Neuwirth C, Petri S, Probst-Schendzielorz K, Pühringer M, Steinbach R, Schara-Schmidt U, Schimmel M, Schrank B, Schwartz O, Schlachter K, Schwerin-Nagel A, Schreiber G, Smitka M, Topakian R, Trollmann R, Tuerk M, Theophil M, Rauscher C, Vorgerd M, Walter MC, Weiler M, Weiss C, Wilichowski E, Wurster CD, Wunderlich G, Zeller D, Ziegler A, Kirschner J, Pechmann A; SMArtCARE study group. Vill K, et al. Among authors: petri s. J Neurol. 2024 May;271(5):2787-2797. doi: 10.1007/s00415-024-12188-5. Epub 2024 Feb 27. J Neurol. 2024. PMID: 38409538 Free PMC article.
372 results