Approximately 3-4% of newborns manifest a genetic disease or major birth defect, and about 12% of all mutations reported are caused by nonsense mutation. Non-sense mutations introduce premature stop codons in the reading frame of a gene. When the mutated sequence is translated into a protein, the resulting protein is incomplete and shorter than normal. Consequently, most nonsense mutations result in nonfunctional proteins. Nonsense mutations account for some of the most severe phenotypes in genetic diseases and often have devastating effects in critical target organs. Eloxx lead compound, ELX-02, provides unique opportunity to potentially be the first disease-modifying therapy for treatment of these set of devastating diseases, for which there are no effective treatments.