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Breakthrough drug for rare genetic disease
Radical new gene therapy restores sight to patients with rare eye condition - BBC News
$2.1 million drug approved for toddler with rare genetic disease
WFAA Original: How two children with a rare genetic disease could lead to breakthrough treatments
Breakthrough for Rare Genetic Disorder?
FDA approves Novartis gene therapy for rare genetic disorder
Patient advocacy group paves way for breakthrough gene therapy for genetic disease
Hollywood boy diagnosed with rare genetic disorder after months of uncertainty
FDA Approval Novo Nordisk's Breakthrough Therapy for Rare Genetic Kidney Condition!
Breakthrough drug saves the life of a young Somers girl
Top 7 Medical Breakthroughs of 2023
Parents' experience caring for baby with rare, deadly, genetic disease
