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Screening for lysosomal storage disorders—A clinical perspective

✍ Scribed by Janice M. Fletcher


Publisher
Springer
Year
2006
Tongue
English
Weight
222 KB
Volume
29
Category
Article
ISSN
0141-8955

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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,