Résumé :
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Communication n° 754 Introduction: Facioscapulohumeral muscular dystrophy (FSHD) is one of the most frequent inherited neuromuscular disorder, with autosomal dominant transmission. This disorder is slowly progressive, involving preferentially the muscles of the face and scapular girdle. There is currently no available treatment. Few therapeutic trials of the disorder have been conducted, most of them with beta2-adrenergic agonists with limited positive effects. Objectives: This work consisted in a multicentric placebo-controlled double-blind randomised study in order to evaluate the effects on muscle strength of salbutamol administered over 6 months. Methods: 112 patients were finally included and equally distributed within the two groups. Salbutamol was administered at daily dose of 16 mg (sustained-release capsules, 2x4 mg twice daily) in a discontinuous mode (3 weeks ON, 1 week OFF). Patients were evaluated at the inclusion (M0) then 3 (M3) and 6 months (M6) later. Each evaluation consisted in clinical examination, ECG, biological analyses, Manuel Muscle Testing (MMT), Quantified Muscle Testing (QMT) and functional scores (10 m walking, 4 stairs climbing, Barré, Mingazzini). Results: The treated group presented a significantly higher age than the placebo group. The functional status of both groups was similar at the inclusion. After 6 months of treatment, global MMT scores were not significantly modified in both groups. No changes in functional scores were also observed. QMT scores are still under analysis, but the first muscles functions analysed such as elbow flexion are not significantly changed by the treatment. Profile of adverse events was comparable in both groups. Conclusion: These preliminary results do not show any positive effects of salbutamol on muscle strength. Further detailed analyses are needed to definitely confirm these results. Acknowledgements : This work was supported by the Association Française contre les Myopathies (AFM).
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