
The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time AAV9 gene therapy, as the first treatment for pediatric MPS IIIA (Sanfilippo syndrome type A).
Even at a young age, a child with a serious or disabling health problem might need a blueprint for lifelong care. Creating that plan has become a business that benefits your patients. Participating in a clinical trial allows you to bring innovative treatments and state-of-the-art therapeutics to your patients, as well as necessary care to those whose families cannot otherwise afford it.






