Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.
Loss of a protein called CLN7 causes levels of multiple soluble lysosomal proteins to drop, contributing to the development and progression of Batten disease, a mouse model shows. The study, ... Read more
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, ... Read more
Researchers found, in a new mouse model for Batten disease, that defective synapses in specific brain regions may be the cause of disease symptoms. The study “Defective synaptic transmission ... Read more
Early photosensitivity may be a hallmark of ceroid lipofuscinosis type 2 (CLN2) disease, according to the results of a small study. The study “Photosensitivity is an early marker of neuronal ceroid lipofuscinosis type 2 disease” was published in the ... Read more
Current therapeutics, including gene therapy, that are based on delivery of TTP1 protein directly into the central nervous system of patients with Batten disease may not be enough to ... Read more
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