The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme ...

New review highlights evolving Pompe disease management, from screening to next-generation therapies and monitoring. Read ...

Thanks to advances in imaging and diagnostic technologies, clinicians can now detect many genetic disorders in the womb, ...

Our bodies perform many critical metabolic reactions, using a wide range of enzymes and other molecules. When there is a ...

The approval follows PEGASUS Phase III trial assessing Palynziq efficacy and safety compared to diet alone in adolescents ...

Agency officials promise fast reviews of new treatments while vowing they will not be a “rubber stamp” for the industry. But ...

Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...

A Carlsbad family is sharing their son’s story after the 5-year-old was diagnosed with a rare genetic disorder and began a ...

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

A Malaysian researcher is studying the prospect of prescribing medicine based on a patient's DNA to help them receive safer ...

A Carrboro mother has pulled in close with UNC researchers after watching her 15-year-old son lose basic skills to a devastating genetic disease. Over several years, Cole regressed, losing most of his ...

Prime Medicine Inc. (NASDAQ:PRME) is one of the best new penny stocks to buy. On March 3, Prime Medicine reported its full-year 2025 financial results, highlighting a shift toward its in vivo liver ...