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NPC-09, SA-ER, UX001

Also known as: UX-001

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Company Nobelpharma Co. Ltd.
DescriptionExtended-release oral formulation of sialic acid
Molecular Target
Mechanism of ActionSubstrate replacement therapy
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentPhase II
Standard IndicationMuscular atrophy
Indication DetailsTreat hereditary inclusion body myopathy (HIBM)
Regulatory Designation

U.S. - Orphan Drug (Treat hereditary inclusion body myopathy (HIBM));
EU - Orphan Drug (Treat hereditary inclusion body myopathy (HIBM))


Ultragenyx Pharmaceutical Inc.

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