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

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 ...

Chromosomal abnormalities can include two basic categories: numerical or structural. A structural abnormality can include an alteration to the structure of the chromosome, as the name suggests.

For informational purposes only. Consult a medical professional for advice. Source: Focus Medica. An X-linked genetic disease caused by the deficiency of enzyme iduronate-2-sulfatase. It is caused ...

Denali Therapeutics Inc. DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, tividenofusp alfa (DNL310), for the treatment of individuals with ...