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Daily salbutamol in young patients with SMA type II

Pane, M; Staccioll, S; Messina, S; D'Arnico, A; Pelliccioni, M; Mazzone, ES; Cuttini, M; ... Mercuri, E; + view all (2008) Daily salbutamol in young patients with SMA type II. NEUROMUSCULAR DISORD , 18 (7) 536 - 540. 10.1016/j.nmd.2008.05.004.

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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. (c) 2008 Elsevier B.V. All rights reserved.

Type: Article
Title: Daily salbutamol in young patients with SMA type II
DOI: 10.1016/j.nmd.2008.05.004
Keywords: spinal muscular atrophy, salbutamol, therapy, children, beta 2 agonists, SPINAL MUSCULAR-ATROPHY, FUNCTIONAL MOTOR SCALE, PILOT TRIAL, MUSCLE STRENGTH, SKELETAL-MUSCLE, ALBUTEROL, AGONIST, INJURY
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Pop Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Pop Health Sciences > UCL GOS Institute of Child Health > ICH Developmental Neurosciences Prog
URI: http://discovery.ucl.ac.uk/id/eprint/114105
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