Novel Mucopolysaccharidosis Tx Designated Orphan Drug

The compound is currently being studied in a Phase 1 first-in-human clinical study
The compound is currently being studied in a Phase 1 first-in-human clinical study

Eloxx announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to ELX-02, its propriety drug candidate for the treatment of mucopolysaccharidosis type 1 (MPS I).

ELX-02 is a synthetic designer aminoglycoside optimized as a translational read-through drug (TRID) for the treatment of genetic diseases caused by nonsense mutations. The compound is being studied in a Phase 1 first-in-human clinical study in healthy volunteers. 

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Mucopolysaccharidosis type I (MPS I) is a chronic, progressive genetic disorder caused by an enzymatic deficiency of alpha-L-iduronidase (IDUA). This disrupts the glycosaminoglycan (GAG) catabolic pathway leading to an intra-lysosomal accumulation of substrates, heparan sulfate (HS) and dermatan sulfate (DS).

For more information call (781) 557-6639 or visit Eloxxpharma.com.

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