Growing up with a rare genetic disorder, Pam Kowalczyk often felt like no one truly understood her daily struggles. Years ...

SAN DIEGO, April 04, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (TVTX), today announced that the Company will present eight posters in classical homocystinuria (HCU) at Society for Inherited ...

Over the next two years, the planned funding will be allocated to projects that have received some FDA feedback as well as emerging efforts.

On Wednesday, Applied Therapeutics, Inc. (NASDAQ:APLT) stock traded higher with a session volume of 31.75 million, compared to an average volume of 1.26 million, according to data from Benzinga Pro.

Initial trial results suggest that a new mRNA therapy may be able to safely and effectively treat propionic acidemia, a rare metabolic disorder. When you purchase through links on our site, we may ...

FutureWise Research, a leading provider of healthcare market intelligence, today announced the release of its comprehensive ...

South Dakota infant Pruitt Kruse became extremely sick with a rare, inherited metabolic disorder and died at just 6 months old. Jordan Kruse realized something was wrong with her newborn, Pruitt, ...

In a global first, scientists have used advanced base editing to repair a deadly gene mutation in a newborn, marking a major leap forward in the treatment of rare ...

Is Obesity Genetic or Environmental? Get All the Details This article was reviewed by Lynn Marie Morski, MD, JD. Key ...

A significant overlap has been identified in the functionalities of genes associated with immune and metabolic disorders, suggesting a fundamental frameshift is needed in our understanding of the two.

The blueprint of who we are begins with the genes passed down from our parents. While these inherited traits give us our eye color and height, they can also contain instructions that increase our risk ...