Newborn screening is a fundamental right for every child, crucial for early diagnosis and intervention of preventable ...

"We are pleased with the results from our investigator-initiated trial, as they show great promise for NGGT002 to serve as a safe and effective treatment for PKU," said Yiting Liu, Ph.D., VP of ...

as they show great promise for NGGT002 to serve as a safe and effective treatment for PKU," said Yiting Liu, Ph.D., VP of Translational Research at NGGT. "With our initial 40-week span of positive ...

(“Next Generation Gene Therapeutics” or “NGGT”), a clinical-stage biotechnology company pioneering the next wave of gene therapy innovation for genetic rare diseases, today announced positive new data ...

NGGT has initiated Phase I/II clinical trials in the U.S. and China to evaluate the safety and efficacy of NGGT002 for the treatment of Phenylketonuria WALNUT CREEK, Calif., Nov. 11, 2024 ...

Clinical Trial Success at Cincinnati Children's Supports Recent US FDA Approval for First Therapy to Directly Replace a Gene ...

The country boasts 15 specialized metabolic disorder clinics, primarily located in urban centers such as Buenos Aires and ...

and identifying markers that might allow us to ‘predict’ babies at particular risk would be an enormous advantage, since we could then target specific surveillance and treatment of such ...

You might be more familiar with the term 'heel prick test' as it was formerly known, but the 'newborn blood spot test' is ...

For newborns with HCM, the preferred first-line treatment is Propranolol, a class of medication known as a beta-blocker (a type of medication that slows down the heart). If hypertrophic cardiomyopathy ...