Maalouf, Katia E.; Makoukji, Joelle; Saab, Sara; Makhoul, Nadine J.; Carmona, Angelica V.; Kinarivala, Nihar; Ghanem, Noël; Trippier, Paul C.; Boustany, Rose Mary Naaman
(NLM (Medline), 2020)
CLN3 disease is a fatal neurodegenerative disorder affecting children. Hallmarks include brain atrophy, accelerated neuronal apoptosis, and ceramide elevation. Treatment regimens are supportive, highlighting the importance ...