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Company BioBlast Pharma Ltd.
DescriptionIntrathecal formulation of azithromycin
Molecular Target Survival of motor neuron 2 centromeric (SMN2)
Mechanism of Action 
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentPreclinical
Standard IndicationSpinal muscular atrophy (SMA)
Indication DetailsTreat spinal muscular atrophy (SMA)
Regulatory Designation

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