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Page 1
Gene therapy for spinal muscular atrophy: timing is key.
Servais L. Servais L. Lancet Reg Health Eur. 2024 Oct 21;47:101112. doi: 10.1016/j.lanepe.2024.101112. eCollection 2024 Dec. Lancet Reg Health Eur. 2024. PMID: 39502079 Free PMC article. No abstract available.
Quantitative proteome analysis of LAP1-deficient human fibroblasts: A pilot approach for predicting the signaling pathways deregulated in LAP1-associated diseases.
Pereira CD, Espadas G, Martins F, Bertrand AT, Servais L, Sabidó E, Chevalier P, da Cruz E Silva OAB, Rebelo S. Pereira CD, et al. Among authors: servais l. Biochem Biophys Rep. 2024 Jun 26;39:101757. doi: 10.1016/j.bbrep.2024.101757. eCollection 2024 Sep. Biochem Biophys Rep. 2024. PMID: 39035020 Free PMC article.
Treatments for RYR1-related disorders.
Raga S, Voermans N, Perez-Neri I, Dowling J, Jungbluth H, Baranello G, Servais L, Tillema A, Wilmshurst J. Raga S, et al. Among authors: servais l. Cochrane Database Syst Rev. 2024 Dec 10;12(12):CD014439. doi: 10.1002/14651858.CD014439. Cochrane Database Syst Rev. 2024. PMID: 39655671 Free PMC article.
Treatment strategies for patients with spinal muscular atrophy.
De Waele L, Servais L. De Waele L, et al. Among authors: servais l. Expert Rev Neurother. 2024 Dec 10:1-3. doi: 10.1080/14737175.2024.2439486. Online ahead of print. Expert Rev Neurother. 2024. PMID: 39648756 Free article. No abstract available.
Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpoint.
Servais L, Strijbos P, Poleur M, Mirea A, Butoianu N, Sansone VA, Vuillerot C, Schara-Schmidt U, Scoto M, Seferian AM, Previtali SC, Tulinius M, Nascimento A, Furlong P, Singh T, Dreghici RD, Goemans N, Mercuri E, Straub V, Ormazabal MG, Braid J, Muntoni F, Tricot A, Annoussamy M, Eggenspieler D. Servais L, et al. Sci Rep. 2024 Nov 29;14(1):29681. doi: 10.1038/s41598-024-80177-9. Sci Rep. 2024. PMID: 39613806 Free PMC article.
280 results