Marissa Russo is a former STAT intern supported by the AAAS Mass Media Science & Engineering Fellowship. In what experts are calling a “dream come true,” scientists used a recent biochemical discovery ...

Iron deficiency anemia occurs when there are insufficient red blood cells due to a lack of iron. In some cases, iron deficiency anemia can be genetic, as people can pass genetic variations onto their ...

ECUR-506 is an investigational gene editing therapy that utilizes an adeno-associated virus to deliver a functional copy of the OTC gene. The Food and Drug Administration (FDA) has granted Fast Track ...

WARREN, N.J., Nov. 13, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment ...

Eladocagene exuparvovec is the first US-approved gene therapy for AADC deficiency, administered directly to the brain. Clinical trials showed significant motor and cognitive improvements, especially ...

In a world with an ageing global population, we could all use some good news when it comes to age-related health. The latest findings in a study led by Dr Feng Hefeng at the Luxembourg Institute of ...