US FDA approves Mirum Pharma's genetic disorder drug
The U.S. Food and Drug Administration approved Mirum Pharmaceuticals' genetic disorder drug on Friday, making it the first ...
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Characterizing the spectrum of mitochondrial DNA deletion disease syndromes
Researchers from the Mitochondrial Medicine Program at Children's Hospital of Philadelphia (CHOP) have better characterized a spectrum of what were classically considered discrete mitochondrial DNA ...
Researchers identify novel gene behind neurodevelopmental syndrome for enhanced clinical treatment
A clinical research team from the LKS Faculty of Medicine, the University of Hong Kong (HKUMed), and international genetic researchers led a global research study using multi-omics analysis and ...
How genetics shape blood proteins during development from childhood to adolescence
Blood proteins serve as crucial indicators of health and disease risk throughout development. Now, researchers at the ...
Scientists Just Found a Cholesterol Clue That Could Change Parkinson’s Treatment
A breakthrough study has uncovered a hidden player in Parkinson’s disease—24-OHC, a cholesterol metabolite that fuels the ...