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Khondrion Phase 3 KHENERFIN study of sonlicromanol in m.3243A>G primary mitochondrial disease, a representative image of rare disease drug development and mitochondrial therapy research.

Pharma & Biotech

Khondrion moves sonlicromanol into Phase 3, but can KHENERFIN finally crack mitochondrial disease?

by Pallavi Madhiraju
April 20, 2026
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Khondrion has dosed the first patient in Phase 3 KHENERFIN for sonlicromanol. Read what it could change in mitochondrial disease treatment.

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Pharma & Biotech

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Representative image of a clinician reviewing skeletal imaging, illustrating Mirum Pharmaceuticals and Incyte’s zilurgisertib Phase 2 progress in fibrodysplasia ossificans progressiva and the rare disease treatment questions facing regulators.

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Representative image of biopharma laboratory research. Gilead Sciences and Merck’s latest Phase 3 updates highlight the promise of once-weekly HIV treatment with islatravir and lenacapavir, while raising fresh questions over Trodelvy’s lung cancer expansion strategy.

Pharma & Biotech

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clinical trials Eli Lilly and Company European Medicines Agency FDA FDA approval immuno-oncology immunotherapy Inc. Johnson & Johnson medical devices oncology precision oncology rare disease U.S. Food and Drug Administration United States Food and Drug Administration

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