Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
Long-term treatment with Kuvan (sapropterin dihydrochloride) helped to control blood levels of phenylalanine, and preserve normal intelligence and growth in infants and children with phenylketonuria (PKU), a Phase 3 trial that ... Read more
Synlogic is recruiting patients for a proof-of-concept Phase 2 trial that is investigating SYNB1618, its investigational treatment for phenylketonuria (PKU), in adults with the disease. The open-label trial (NCT04534842), initially ... Read more
Dr. Reddy’s Laboratories has launched a generic version of BioMarin Pharmaceuticals’ Kuvan for the treatment of adults and children with phenylketonuria (PKU) in the U.S. This generic will be distributed ... Read more
Compared with conventional low-phenylalanine protein substitutes, a prolonged-release protein substitute caused fewer gastrointestinal problems in children and adolescents with phenylketonuria (PKU), a study has found. The study, “An Observational Study ... Read more
A single dose of the gene-editing construct used in Homology Medicines‘ investigational therapy for phenylketonuria (PKU) in children, shows high efficiency and precision in inserting a fully functional copy of ... Read more
Healthcare services need to be restructured in Italy to better support adults with phenylketonuria (PKU), regardless of whether or not they follow strict diet regimens, a study suggests. The study, ... Read more
The first patient in a Phase 1b clinical trial of Rubius Therapeutics‘ RTX-134, an experimental cell therapy for phenylketonuriara, has received a dose of the treatment, the company said. The open-label ... Read more