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Expert opinion on orphan drugs |
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Article
Objective: Advanced Therapy Medicinal Products (ATMPs) present significant therapeutic advantages for inherited rare diseases. However, the development of orphan ATMPs is challenging due to their complexity and unpredictable biological activity.[...]Article
Matsuo M | 2015Introduction: Becker muscular dystrophy (BMD) is a progressive X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Duchenne muscular dystrophy (DMD) is also caused by such mutations but shows rapidly progressive muscle wa[...]