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• W2007183994 abstract "The aim of this open pilot study was to establish the profile of tolerability and clinical response of salbutamol (albuterol) in a cohort of young children affected by type II spinal muscular atrophy (SMA). Twenty-three children between 30 months and 6 years of age were treated with salbutamol (2 mg three times a day) for 1 year. All children were longitudinally assessed using the Hammersmith motor functional scale 6 months before treatment started (T0), at baseline (T1) and 6 and 12 months later. There was no significant change in function between T0 and T1 assessments, but the functional scores recorded after 6 and 12 months of treatment were significantly higher than those recorded at baseline (p=0.006). Our results suggest that salbutamol may be beneficial to SMA patients without producing any major side effect. Larger prospective randomized, double-blind, placebo controlled trials are needed to confirm these preliminary findings." @default.
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• W2007183994 date "2008-07-01" @default.
• W2007183994 modified "2023-10-18" @default.
• W2007183994 title "Daily salbutamol in young patients with SMA type II" @default.
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• W2007183994 doi "https://doi.org/10.1016/j.nmd.2008.05.004" @default.
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