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LJPC-0712

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Company La Jolla Pharmaceutical Co.
DescriptionFormulation of the GABA A neuromodulator allopregnanolone
Molecular Target GABA A receptor
Mechanism of ActionGABA A receptor modulator
Therapeutic Modality 
Latest Stage of DevelopmentPreclinical
Standard IndicationGlycosphingolipid storage disorders
Indication DetailsTreat Niemann-Pick disease type C1 (NPC1)
Regulatory Designation

U.S. - Orphan Drug (Treat Niemann-Pick disease type C1 (NPC1))

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