The autosomal dominant optic atrophy treatment market is experiencing significant expansion, driven by increasing disease awareness, rising prevalence, and R&D in precision medicine from key ...

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Such a cobblestone tongue is a telltale sign of a rare genetic condition called Cowden syndrome. It's caused by inherited ...

Ultimately, Stoke Therapeutics is that classic “intriguing biotech” --hurdling to one of those proverbial “moments of truth” ...

A large longitudinal study found that pesticide exposure and caffeinated soda intake were linked to more severe motor ...

The well-studied NF-κB signaling system is a key mediator of the inflammatory response. Large-scale sequencing studies in ...

Huntington’s disease is a rare genetic disorder affecting movement, memory, and mood. Here’s what we know—and what’s ahead.

Hereditary AE results from inherited zinc malabsorption, often due to mutations in the SLC39A4 gene. It responds well to zinc supplementation, which usually requires lifelong treatment. Acquired AE is ...