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genetic diseases
Research
Study reveals expanding gene that causes Huntington's disease
The disease-causing variant of the gene slowly expands over time until it hits a toxic threshold that leads to nerve cell death.
Darren Incorvaia
Jan 17, 2025 5:00pm
BioMarin goes CAMPing, striking RNA deal with biotech
Oct 1, 2024 9:55am
'Clinical intuition' led FDA advisors to back Zevra's rare disease med
Aug 5, 2024 10:35am
Ultragenyx, Mereo's brittle bone drug sustains fracture rate cut
Jun 12, 2024 8:55am
Avidity therapy shows possible benefit in muscular dystrophy
Jun 12, 2024 7:00am
Otsuka pays Ionis $65M for European rights to rare disease drug
Dec 19, 2023 10:18am