At Minoryx, we are committed to finding innovative treatments for life threatening rare diseases.
We are currently focused on Inborn Errors of Metabolism, a group of rare diseases of genetic origin with highly unmet medical need. Such diseases are life threatening and highly debilitating, usually with neurological manifestations and affect most severely the pediatric population.
We develop a new generation of pharmacological chaperones, a small molecule drug class aimed to restore protein functionality and which offer one of the most promising approaches to the treatment of genetic diseases severely affecting the central nervous system. As a complementary approach, Minoryx is also involved on developing small molecule drugs which counteract the cellular dysfunctions originated by the corresponding protein deficiency.
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