Sherri Gordon, CLC is a certified professional life coach, author, and journalist covering health and wellness, social issues, parenting, and mental health. She also has a certificate of completion ...
An Applied Therapeutics drug widely expected to secure FDA approval has been turned down by the agency, a setback for the company’s bid to bring to market the first therapy for a rare inherited ...
Advisory Committee meeting would no longer be required, which was previously tentatively scheduled for October 9. The previously announced Prescription Drug User Fee Act target action date remains on ...
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 pivotal medical milestone has been reached. For the first time ever, researchers have used a personalized CRISPR-based gene therapy to treat an infant’s rare and life-threatening illness. Doctors at ...
SAN DIEGO, April 04, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present eight posters in classical homocystinuria (HCU) at Society for ...
A new study, published in Nature Genetics, created the largest genetic map of human metabolism, revealing new insights into ...
Half of all babies diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency die within their first week of life Getty KJ Muldoon was diagnosed with severe carbamoyl phosphate ...
Surgeons at Children's Healthcare of Atlanta performed the world's youngest domino liver transplant. Domino liver transplants involve one patient with ...
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.
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