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metachromatic leukodystrophy
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Platelet degranulation
species not specified
one gene
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PSAP
Crystal structure of saposin B reveals a dimeric shell for lipid binding
ARSA
Enzyme, cell and gene-based therapies for metachromatic leukodystrophy
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Clinical Trials
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External
A Safety and Efficacy Study of Cryopreserved GSK2696274 for Treatment of Metachromatic Leukodystrophy (MLD)