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Gene Therapy Services In Netherlands

1 services found
Oral Treatment for Lysosomal Storage Disorders

Oral Treatment for Lysosomal Storage Disorders

by:Azafaros   based inNaarden, NETHERLANDS
Oral administration allows for home-based, lifelong treatment, enhancing quality of life, particularly for pediatric patients, and reducing dependency on gene or enzyme replacement therapies. The U.S. FDA's Orphan Drug Designations underscore its potential. Preclinical studies further suggest its broader applicability in lysosomal storage disorders by ...
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