Posts by: Joana Carvalho

Joana Carvalho

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.

February 3, 2021February 3, 2021

Kuvan Safely Aids Growth, Intellect of Youngest Patients Over Long Term, Trial Finds

News

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

January 27, 2021January 27, 2021

Synlogic Recruiting Adult Patients for Phase 2 Trial of SYNB1618

News

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

October 21, 2020October 21, 2020

Dr. Reddy’s Launches Generic of Kuvan for PKU Patients in the US

News

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

September 23, 2020September 23, 2020

Prolonged-Release Protein Substitute Seen to Cause Fewer GI Problems in PKU Children

News

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

May 20, 2020May 20, 2020

Homology’s Potential Gene-editing Strategy for PKU Shows Promise in Animal Model

News

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

May 6, 2020May 6, 2020

Study Calls for Healthcare Restructuring in Italy to Better Suit Needs of Adults With PKU

News

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

February 5, 2020February 5, 2020

First Dose Given in Phase 1b Trial of Rubius’ RTX-134 for Phenylketonuria

News

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