The U.S. Food and Drug Administration (FDA) accepted Abeona Therapeutics’ investigational new drug application for its one-time gene…
Articles by José Lopes, PhD
Treatment with Brineura (cerliponase alfa) reduces decline in motor and language function in children with ceroid lipofuscinosis type…
Gene therapy is able to reduce toxic protein aggregates in a zebrafish model of one form of adult-onset Batten disease,…
Stem cell models of lysosomal storage disorders (LSDs), including Batten disease (BD), could significantly…
Lopid (gemfibrozil), an FDA-approved lipid-lowering drug, was found to improve life expectancy and motor function in mice with late infantile neuronal…