Abeona Therapeutics’ one-time gene therapy candidate ABO-202 has been granted fast track designation by the U.S. Food and Drug Administration (FDA) for treating…
Articles by Patricia Inácio, PhD
The U.S. Food and Drug Administration has granted rare pediatric disease designation to Regenxbio’s one-time gene therapy RGX-181 for the treatment of late-infantile…
The activation of a recycling pathway, a process called autophagy, which leads to large storage vesicles in muscle cells, is…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Regenxbio’s one-time gene therapy RGX-181 for the treatment of…
Regenxbio is developing a novel, one-time gene therapy, called RGX-181, for the treatment of late-infantile neuronal ceroid…
Husbandry conditions of mouse models of CLN3-Batten disease may influence research outcomes, such as potential disease biomarkers, a new…
The expression of two genes associated with neuronal ceroid lipofuscinosis, better known as Batten disease, was restricted to certain cells of…
Long-term follow-up of two siblings with Batten disease unveiled symptoms of slowly progressive motor deterioration, persistent photosensitivity, and seizures without…
Loss of a protein called CLN7 causes levels of multiple soluble lysosomal proteins to drop, contributing to the development and progression…
The U.S. Food and Drug Administration (FDA) has granted a rare pediatric disease designation to Abeona Therapeutics’ investigational ABO-202…