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.
Swedish Orphan Biovitrum AB’s (Sobi) has dosed the first patient in its Phase 1/2 clinical trial evaluating SOBI003 to treat Sanfilippo syndrome type A. The SOBI003-001 study (NCT03423186), which is still recruiting participants, ... Read more
High numbers of antibodies targeting different types of adeno-associated viruses (AAV) pose challenges for AAV-based gene therapies in Sanfilippo syndrome children, according to researchers. The study, “Differential prevalence of ... Read more
A mouse model for Sanfilippo Syndrome type B recreates the main characteristics of humans with the disease, indicating a potential use for therapeutic approaches. The study with that finding, ... Read more
Researchers have developed and characterized the first fruit fly model for Sanfilippo syndrome to help advance knowledge about the cellular and molecular mechanisms that drive symptoms of the condition. ... Read more
Mild to moderate hearing loss is common among children and young adults with different mucopolysaccharidosis (MPS) diseases, including Sanfilippo syndrome, a small study reports. The study, “Audiometric evaluation in individuals ... Read more
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