The FDA has approved Ctexli for the treatment of cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease, in ...
The U.S. Food and Drug Administration approved Mirum Pharmaceuticals' genetic disorder drug on Friday, making it the first ...
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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 ...
Neurofilament light (NFL) is a powerful and sensitive biomarker for tracking neuronal health and disease progression.
Qure's AMT-130 shows 80% Huntington's progress reduction, eyeing FDA approval. Click here to read why I rate QURE stock a Buy ...
Researchers from the Huntington's Disease Center, UCL Queen Square Institute of Neurology, and UK DRI at UCL have taken major ...
A new discovery offers hope for Huntington’s disease. This discovery provides hope that a DNA repair process may help slow or ...
Four more experimental drugs for amyotrophic lateral sclerosis (ALS) have failed to hit the primary endpoint in the phase 2/3 ...
U.S. President Donald Trump's administration needs to speed up generic drug approvals and exempt some drugs from funding ...
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