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Ace-ER, NPC-09 (formerly UX001)

Also known as: UX-001 (formerly SA-ER)

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Company Nobelpharma Co. Ltd.
DescriptionExtended-release oral formulation of aceneuramic acid
Molecular Target
Mechanism of ActionSubstrate replacement therapy
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentPhase III
Standard IndicationMuscular atrophy
Indication DetailsTreat GNE myopathy (GNEM)/hereditary inclusion body myopathy (HIBM)
Regulatory Designation U.S. - Orphan Drug (Treat GNE myopathy (GNEM)/hereditary inclusion body myopathy (HIBM));
EU - Orphan Drug (Treat GNE myopathy (GNEM)/hereditary inclusion body myopathy (HIBM))
Partner Ultragenyx Pharmaceutical Inc.

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