Long-Term Efficacy Data in Rare Disease Gene Therapy Programs
Long-Term Efficacy Data in Rare Disease Gene Therapy Programs Assessing Long-Term Efficacy in Gene Therapy for Rare Diseases Introduction: Why Long-Term Data Matters in Gene Therapy Gene therapy has emerged as a transformative treatment for rare diseases, offering the possibility of one-time interventions that deliver lasting clinical benefit. However, regulators, clinicians, and patients alike require…
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