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Observational study of spinal muscular atrophy type I and implications for clinical trials.
Finkel RS, McDermott MP, Kaufmann P, Darras BT, Chung WK, Sproule DM, Kang PB, Foley AR, Yang ML, Martens WB, Oskoui M, Glanzman AM, Flickinger J, Montes J, Dunaway S, O'Hagen J, Quigley J, Riley S, Benton M, Ryan PA, Montgomery M, Marra J, Gooch C, De Vivo DC. Finkel RS, et al. Neurology. 2014 Aug 26;83(9):810-7. doi: 10.1212/WNL.0000000000000741. Epub 2014 Jul 30. Neurology. 2014. PMID: 25080519 Free PMC article.
OBJECTIVES: Prospective cohort study to characterize the clinical features and course of spinal muscular atrophy type I (SMA-I). METHODS: Patients were enrolled at 3 study sites and followed for up to 36 months with serial clinical, motor functi …
OBJECTIVES: Prospective cohort study to characterize the clinical features and course of spinal muscular atrophy typ …
Can the CHOP-INTEND be used as An Outcome Measure in the First Months of Age? Implications for Clinical Trials and Real World Data.
Cutrona C, de Sanctis R, Coratti G, Capasso A, Ricci M, Stanca G, Carnicella S, Utlulig M, Bersani G, Lazzareschi I, Leoni C, Buonsenso D, Luciano R, Vento G, Finkel RS, Pane M, Mercuri E. Cutrona C, et al. J Neuromuscul Dis. 2024;11(1):85-90. doi: 10.3233/JND-221644. J Neuromuscul Dis. 2024. PMID: 37980678 Free PMC article.
OBJECTIVE: The aim of our study was to assess the maturation of the CHOP-INTEND scores in a group of healthy infants, establishing which items of the scale can be reliably used in individuals younger than 3 months. METHODS: This is a prospective observational study. …
OBJECTIVE: The aim of our study was to assess the maturation of the CHOP-INTEND scores in a group of healthy infants, establishing which ite …