Author Archives: Patricia Inacio PhD

Clinical Evolution of Batten Disease in Young Adult Siblings Described in Case Report

Long-term follow-up of two siblings with Batten disease unveiled symptoms of slowly progressive motor deterioration, persistent photosensitivity, and seizures without dementia, according to a case report. The case report, “Long-term follow-up of two siblings with adult-onset neuronal ceroid lipofuscinosis, Kufs type A,” was published in the journal Epileptic Disorders.

FDA Grants Rare Pediatric Disease Designation to ABO-202 for Infantile Batten Disease

The U.S. Food and Drug Administration (FDA) has granted a rare pediatric disease designation to Abeona Therapeutics’ investigational ABO-202 program being developed for Batten disease caused by mutations in the CLN1 gene, also known as infantile neuronal ceroid lipofuscinosis. ABO-202 is an adeno-associated virus (AAV)-based gene therapy (AAV-CLN1) where weakened…