FDA approves 1st treatment for rare childhood genetic disease

The FDA has approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome type A. Fayuvi is a one-time intravenous gene therapy that uses adeno-associated virus serotype 9 to deliver a working copy of the SGSH gene into patients’ cells, according to a Sept. 17 news […] The post FDA approves 1st treatment for rare childhood genetic disease appeared first on Becker's Hospital Re...