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Explore conditions, clinical trials, and the organisations running them.
Up to: Inborn errors of metabolism
Lysosomal storage disease
A metabolic disorder caused by mutations in proteins critical for lysosomal function, including lysosomal enzymes, lysosomal integral membrane proteins, and proteins involved in the post-translational modification and trafficking of lysosomal proteins.
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Lysosomal lipid storage disorder 2 trials · 192 incl. sub-types Sub-types →
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Mucopolysaccharidosis 14 trials · 61 incl. sub-types Sub-types →
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Lysosomal glycogen storage disease 0 trials · 45 incl. sub-types Sub-types →
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Inborn disorder of lysosomal amino acid transport 0 trials · 15 incl. sub-types Sub-types →
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Glycoproteinosis 0 trials · 14 incl. sub-types Sub-types →
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Late infantile neuronal ceroid lipofuscinosis 1 trial · 5 incl. sub-types Sub-types →
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Disorder of sialic acid metabolism 0 trials · 1 incl. sub-types Sub-types →
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Glycoprotein storage disease 0 trials
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Hereditary spastic paraplegia 48 0 trials
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Pycnodysostosis 0 trials