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Minoryx: Mediating rare metabolism

Minoryx's chaperones and repurposed drugs treat inherited metabolic diseases

Minoryx Therapeutics S.L. has two complementary strategies for developing therapies to treat inherited metabolic diseases involving enzyme deficiencies: identifying small molecule chaperones that can restore mutant enzyme function, and repurposing existing drugs to treat the effects of enzyme deficiency when restoring function is not an option.

Minoryx expects its lead compound will treat a different subset of X-linked adrenoleukodystrophy patients than the lone product in the clinic for the Orphan indication.

In inherited metabolic diseases, genetic mutations produce unstable or dysfunctional enzymes that cells

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