Despite having gone through this before with her first son, Yusof, and despite the doctors explaining that this was a gene ...

With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now ...

Created with Sketch. This rare sex chromosome abnormality, also referred to as Jacob’s syndrome, occurs when a male infant is born with an extra Y chromosome. Some symptoms include being tall ...

The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder. Five ...

Women are born with two X chromosomes and inherit one from each parent. But in every cell of their body, just one X chromosome is needed – so the other is randomly inactivated. Some cells use only a ...

Most people carry two "versions" of the first 22 chromosomes ... as far back as when humans were hunter-gatherers. The six individuals we identified with Down syndrome were all from Europe ...

1,2 Numerical abnormalities are more common than structural abnormalities, with these mutations in sex chromosomes having different implications compared to autosomes. 2 Klinefelter’s syndrome ...

Denali’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).