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Increase in use of gene therapy for rare diseases
Focus on Rare Disease: Prime time – gene therapy for rare disorders
The Challenge to Develop and Commercialize Gene-Based Therapies for Rare Diseases
The Promises and Pitfalls of Gene Therapy for Rare Diseases
FDA approves Novartis gene therapy for rare genetic disorder
Advancing the Development of Gene Therapy for Rare Diseases Center for Individualized Medicine 2024
The Promises and Pitfalls of Gene Therapy for Rare Diseases: Strategies for Maximizing Reg Success
Gene Therapy and Platform Approaches to Treating Rare Diseases
Genomics Lite: Why are some genes dominant?
Challenges in Rare Disease Treatment
Radical new gene therapy restores sight to patients with rare eye condition - BBC News
Grand Rounds: Developing Gene Therapies for Ultra-rare Diseases in the Academic Setting
