Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study

BACKGROUND: Pompe disease is a rare metabolic myopathy for which disease-specific enzyme replacement therapy (ERT) has been available since 2006. ERT has shown efficacy concerning muscle strength and pulmonary function in adult patients. However, no data on the effect of ERT on the survival of adult...

全面介紹

Na minha lista:
書目詳細資料
Main Authors: Güngör, Deniz, Kruijshaar, Michelle E, Plug, Iris, D’Agostino, Ralph B, Hagemans, Marloes LC, van Doorn, Pieter A, Reuser, Arnold JJ, van der Ploeg, Ans T
格式: Artigo
語言:Inglês
出版: BioMed Central 2013
主題:
Research
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC3623847/
https://ncbi.nlm.nih.gov/pubmed/23531252
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-8-49
標簽: 添加標簽
沒有標簽, 成為第一個標記此記錄!

相似書籍