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arimoclomol (OR-04)

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Company Orphazyme ApS
DescriptionOral small molecule heat shock response inducer
Molecular Target Heat shock protein 70 (Hsp70)
Mechanism of Action 
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentPhase II/III
Standard IndicationAmyotrophic lateral sclerosis (ALS)
Indication DetailsTreat superoxide dismutase 1 (SOD1) linked familial amyotrophic lateral sclerosis (FALS)
Regulatory Designation

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