Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis

Author:

Schoser BenediktORCID,Stewart Andrew,Kanters Steve,Hamed Alaa,Jansen Jeroen,Chan Keith,Karamouzian Mohammad,Toscano Antonio

Publisher

Springer Science and Business Media LLC

Subject

Neurology (clinical),Neurology

Reference41 articles.

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2. Hirschhorn R, Reuser A (2001) Glycogen storage disease Type II: acid alpha-glucosidase (acid maltase) deficiency. In: Scriver C, Beaudet A, Sly W, Valle D (eds) The metabolic and molecular bases of inherited disease, 8th edn. McGraw-Hill, New York, pp 3389–3420

3. Lashley FR (2005) Clinical genetics in nursing practice, 3rd edn. Springer Publishing Company Inc., New York

4. Meikle PJ, Hopwood JJ, Clague AE, Carey WF (1999) Prevalence of lysosomal storage disorders. JAMA 281(3):249–254

5. Hagemans ML, Winkel LP, Hop WC, Reuser AJ, Van Doorn PA, Van der Ploeg AT (2005) Disease severity in children and adults with Pompe disease related to age and disease duration. Neurology 64(12):2139–2141

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