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Page 1
MicroRNAs as serum biomarkers in Becker muscular dystrophy.
Gagliardi D, Rizzuti M, Brusa R, Ripolone M, Zanotti S, Minuti E, Parente V, Dioni L, Cazzaniga S, Bettica P, Bresolin N, Comi GP, Corti S, Magri F, Velardo D. Gagliardi D, et al. Among authors: minuti e. J Cell Mol Med. 2022 Sep;26(17):4678-4685. doi: 10.1111/jcmm.17462. Epub 2022 Jul 26. J Cell Mol Med. 2022. PMID: 35880500 Free PMC article.
Risdiplam in Type 1 Spinal Muscular Atrophy.
Baranello G, Darras BT, Day JW, Deconinck N, Klein A, Masson R, Mercuri E, Rose K, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Seabrook T, Fontoura P, Servais L; FIREFISH Working Group. Baranello G, et al. N Engl J Med. 2021 Mar 11;384(10):915-923. doi: 10.1056/NEJMoa2009965. Epub 2021 Feb 24. N Engl J Med. 2021. PMID: 33626251 Clinical Trial.
Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.
Darras BT, Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Xiong H, Zanoteli E, Baranello G, Bruno C, Vlodavets D, Wang Y, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Fontoura P, Servais L; FIREFISH Working Group. Darras BT, et al. N Engl J Med. 2021 Jul 29;385(5):427-435. doi: 10.1056/NEJMoa2102047. N Engl J Med. 2021. PMID: 34320287