Rare & Genetic Diseases

| 4 min read
In a Phase 2b trial, Khondrion’s sonlicromanol led to significant health benefits for patients with mitochondrial disease linked to a common mutation. 
| 3 min read
A gene therapy targeting organs beyond the brain could improve quality of life and possibly extend survival in children with the neurodegenerative disease.Page 1 of 19 - 374 Total Items


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