Genetic variant analysis indicated that over 70% of subjects had genotypes consistent with classical PKU, underscoring the treatment’s potential across different patient segments. Sepiapterin ...

PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics ...

Genetic variant analysis indicated that over 70% of subjects had genotypes consistent with classical PKU, underscoring the treatment’s potential across different patient segments. Sepiapterin, known ...

PTC Therapeutics (PTCT) shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 ...

A new study suggests early detection improves outcomes, but metabolic episodes in the first days of life still prove challenging.

Additionally, genetic variant analysis of subjects participating in the APHENITY study demonstrates that over 70 percent had a Genotype-Phenotype Value consistent with classical PKU. The ...

BioMarin (BMRN) announced positive new data from studies of Voxzogo in children with achondroplasia and in ongoing clinical trials ...

Atlanta-based workers are baffled by Trump administration cuts to research that directly supports departmental health improvement goals.

Learn more about whether PTC Therapeutics, Inc. or Sarepta Therapeutics, Inc. is a better investment based on AAII's A+ ...

Effie did her PhD studies in Athens on mechanisms of stochastic gene expression. In 2009 ... and Ph.D. in Biochemistry and Molecular Biology from Peking University (PKU) and performed postdoctoral ...

The survey also identified Nagchu as a habitat for at least six first-class protected species in China, including the Tibetan ...