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RG7800 tetrahydrochloride

RG7800 hydrochloride is an orally bioavailable small molecule that potently modulates SMN2 splicing (EC50 = 23 nM) and increases SMN protein levels (EC50 = 87 nM) . It is a promising candidate for spinal muscular atrophy research, with demonstrated activity in both cellular assays and preclinical animal models.

Product Specifications

Field of Research

Cell Biology, Molecular Biology

Molecular Formula

C24H32Cl4N6O

Molecular Weight

562.36

Storage Conditions

-20°C

Notes

For research use only.

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