Newborn screening for inborn errors of metabolism (IEMs) is an essential public health initiative that facilitates early detection of genetic disorders affecting metabolic pathways. By utilising ...

The International Congress of Inborn Errors of Metabolism (ICIEM) is a major international conference held every four years, focusing on the study and treatment of inherited metabolic disorders. ICIEM ...

Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company shared two oral presentations in classical homocystinuria (HCU) at the International Congress of Inborn Errors of Metabolism ...

Moderna is a leader in the creation of the field of mRNA medicine. Through the advancement of mRNA technology, Moderna is reimagining how medicines are made and transforming how we treat and prevent ...

GAINESVILLE, Fla.--(BUSINESS WIRE)--Cyclo Therapeutics, Inc. (Nasdaq: CYTH) (“Cyclo Therapeutics” or the “Company”), a clinical stage biotechnology company dedicated to developing life-changing ...

A disorder called hereditary hemochromatosis, caused by a gene mutation, results in the body absorbing too much iron, leading to tissue damage and conditions like liver disease, heart problems and ...

Inherited diseases of metabolism and immunity have more in common than previously recognized, according to a new study. The findings point to a new set of metabolic genes that are important for the ...

In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of ...

CAMBRIDGE, MA / ACCESS Newswire / September 2, 2025 / Moderna, Inc. (NASDAQ:MRNA) today announced that five abstracts on its investigational mRNA therapeutics have been accepted for presentation at ...