Daily salbutamol in young patients with SMA type II

Marika Pane, Susanna Staccioli, Sonia Messina, Adele D'Amico, Marco Pelliccioni, Elena S. Mazzone, Marina Cuttini, Paolo Alfieri, Roberta Battini, Marion Main, Francesco Muntoni, Enrico Bertini, Marcello Villanova, Eugenio Mercuri

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish
Pages (from-to)536-540
Number of pages5
JournalNeuromuscular Disorders
Volume18
Issue number7
DOIs
Publication statusPublished - Jul 2008

Keywords

  • β2 Agonists
  • Children
  • Salbutamol
  • Spinal muscular atrophy
  • Therapy

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health
  • Developmental Neuroscience
  • Neurology

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