Search Results for: ABO-201


ABO-201 is being developed by Abeona Therapeutics as a gene therapy for a form of Batten disease known as juvenile Batten disease. The investigative treatment is at preclinical stages of development. The U.S. Food ... Read more

Batten Disease Prognosis

Batten disease is a rare inherited neurological condition. It affects compartments in the cell called lysosomes, which are responsible for the degradation of substances that are no longer needed. ... Read more

Gene Therapy

Batten disease, also known as neuronal ceroid lipofuscinoses (NCL) is a rare inherited neurodegenerative disorder. The disease affects lysosomes, small compartments in the cell that degrade components that are no ... Read more