Entwicklung neuer Therapien für seltene Erkrankungen
Author:
Publisher
Springer Science and Business Media LLC
Subject
Surgery,Pediatrics, Perinatology and Child Health
Link
http://link.springer.com/content/pdf/10.1007/s00112-016-0231-7.pdf
Reference27 articles.
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2. Cox TM (2015) Innovative treatments for lysosomal diseases. Best Pract Res Clin Endocrinol Metab 29:275–311
3. de Ruijter J, Valstar MJ, Narajczyk M et al (2012) Genistein in Sanfilippo disease: a randomized controlled crossover trial. Ann Neurol 71:110–120
4. Hein LK, Bawden M, Muller VJ et al (2004) Alpha-L-iduronidase premature stop codons and potential read-through in mucopolysaccharidosis type I patients. J Mol Biol 338:453–462
5. Hennermann JB, Gökce S, Solyom A et al (2016) Treatment with pentosan polysulphate in patients with MPS I: results from an open label, randomized, monocentric phase II study. J Inherit Metab Dis 39:831–837
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