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mitochondrial disorders

PharmaDeviceNews mitochondrial disorders
Pretzel Therapeutics advances PX578 as a potential disease-modifying therapy for POLG disease
Pharma & Biotech

Can Pretzel Therapeutics’ PX578 finally address the root cause of POLG disease in mitochondrial DNA depletion syndromes?

Pretzel Therapeutics advances PX578 for POLG disease. Discover how mitochondrial DNA restoration could reshape treatment for rare mitochondrial disorders.

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Pharma & Biotech

What GenSight Biologics’ French CUA decision means for LHON gene therapy

France grants compassionate use access to GenSight Biologics’ LUMEVOQ for LHON. Explore what this decision changes and what risks remain.

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