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Enzyme replacement therapy for Fabry's disease – Authors' reply

✍ Scribed by A Mehta; JTR Clarke; M Beck; P Elliott; R Schiffmann


Book ID
117306643
Publisher
The Lancet
Year
2010
Tongue
English
Weight
55 KB
Volume
375
Category
Article
ISSN
0140-6736

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Fabry disease: enzyme replacement therap
✍ MR Bongiorno; G Pistone; M Aricò 📂 Article 📅 2003 🏛 John Wiley and Sons 🌐 English ⚖ 164 KB

## ABSTRACT Fabry disease is a multisystem disorder associated with wide variability in clinical expression. Fabry disease is an X‐linked lysosomal storage disorder caused by a deficiency of α‐galactosidase A. The enzyme defect leads to the systemic accumulation of glycosphingolipids with α‐galacto