EU approval for Abeona’s gene therapy to treat Sanfilippo Syndrome Type A
The European Medicines Agency (EMA) Committee has granted orphan drug designation for Abeona‘s lead gene therapy program ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA), a rare autosomal recessive disease that causes neurocognitive decline, speech loss, loss of mobility, and premature death in children.
