𝔖 Bobbio Scriptorium
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63. Pharmacological chaperone treatment for lysosomal storage disorders

✍ Scribed by David Lockhart


Book ID
116988248
Publisher
Elsevier Science
Year
2008
Tongue
English
Weight
43 KB
Volume
93
Category
Article
ISSN
1096-7192

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Therapy for lysosomal storage disorders
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## Abstract In the last years, much progress has been achieved in the field of lysosomal storage disorders. In the past, no specific treatment was available for the affected patients; management mainly consisted of supportive care and treatment of complications. As orphan drug regulations, however,