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 ...

Early administration of the drug Fosdenopterin/rcPMP improves the chances of survival of infants with MoCD type A and promotes the development of brain functions. The drug fosdenopterin/rcPMP ...

The MarketWatch News Department was not involved in the creation of this content. Metabolic Disorders Therapeutics Market Poised to Grow from US$74.97 Billion in 2024 to US$157.65 Billion by 2033 | ...

Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

The poster highlights the findings of pharmacological and biochemical studies which demonstrate that mavodelpar (REN001) is a potent and selective agonist of peroxisome proliferator-activated receptor ...

Kerala doctors successfully performed a life-saving liver transplant on a two-and-a-half-year-old child diagnosed with the ...

Gene editing has moved from theory to bedside with a speed that would have seemed impossible a decade ago. A new wave of ...

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 ...

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 ...

Certain rare genetic disorders may cause a child to develop obesity. Health experts may refer to these conditions as syndromic childhood obesity. They may affect a child’s metabolism or cause them to ...

Metabolic disorders and organic acidemias comprise a diverse group of inherited conditions in which enzymatic deficiencies disrupt normal metabolic pathways. Such deficiencies lead to the accumulation ...