Eloxx Pharmaceuticals, Inc. (Nasdaq: ELOX), a clinical-stage biopharmaceutical company developing exaluren for the treatment of rare kidney diseases, today reported financial results for the second ...

Completed underwritten public offering with gross proceeds of $66.0 million and uplisting to the Nasdaq Capital Market, significantly strengthening the Company's ...

The pathogenicity of MECP2 C-terminal deletions is determined by translational reading frame, enabling prediction of clinical outcome and a shared therapeutic genome editing strategy.

Every living cell, from the simplest bacterium to a human neuron, relies on the same basic machinery to read, interpret, and use its DNA. This molecular machinery is so fundamental that life as we ...