Somatropin treatment of spinal muscular atrophy: a placebo-controlled, double-blind crossover pilot study.

@article{Kirschner2014SomatropinTO,
  title={Somatropin treatment of spinal muscular atrophy: a placebo-controlled, double-blind crossover pilot study.},
  author={Janbernd Kirschner and David C Schorling and Dieter Hauschke and Cornelia Rensing-Zimmermann and U Wein and Ulrike I Grieben and Gudrun Schottmann and Ulrike Schara and Katja Konrad and Wolfgang M{\"u}ller-Felber and Simone Thiele and Ekkehard K G Wilichowski and Elke Hobbiebrunken and Georg M. Stettner and Rudolf Korinthenberg},
  journal={Neuromuscular disorders : NMD},
  year={2014},
  volume={24 2},
  pages={134-42}
}
In preclinical studies growth hormone and its primary mediator IGF-1 have shown potential to increase muscle mass and strength. A single patient with spinal muscular atrophy reported benefit after compassionate use of growth hormone. Therefore we evaluated the efficacy and safety of growth hormone treatment for spinal muscular atrophy in a multicenter, randomised, double-blind, placebo-controlled, crossover pilot trial. Patients (n = 19) with type II/III spinal muscular atrophy were randomised… CONTINUE READING
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