Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease
JOURNAL OF NEUROMUSCULAR DISEASES（2024）
关键词
Patient-Focused Drug Development (PFDD),Duchenne Muscular Dystrophy (DMD),Dystrophinopathies,Natural History of DMD,Genetic Testing and Diagnosis,Outcome Measures,Cardiomyopathy In DMD,Gene Therapy for DMD,Patient Experience and Engagement,Regulatory Guidance and Considerations
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