November 21, 2025 review Open access Study Designs and Crafting Endpoints for Gene Therapy Development Programs in Rare Disease: A Narrative Review Advances in Therapy DOI: 10.1007/s12325-025-03385-3 Full text (OA) OpenAlex Authors 0 Author list not loaded yet. Cited by 0 stored of 3 References 1 Search Sort Most cited Newest Oldest Patent citations Title Any typearticle review book-chapter conference-paper preprint dissertation book dataset other Any fieldAgricultural and Biological Sciences Arts and Humanities Biochemistry, Genetics and Molecular Biology Business, Management and Accounting Chemical Engineering Chemistry Computer Science Decision Sciences Dentistry Earth and Planetary Sciences Economics, Econometrics and Finance Energy Engineering Environmental Science Health Professions Immunology and Microbiology Materials Science Mathematics Medicine Neuroscience Nursing Pharmacology, Toxicology and Pharmaceutics Physics and Astronomy Psychology Social Sciences Veterinary Open access A roadmap to using randomization in clinical trials 2021 BMC Medical Research Methodology article Mathematics Statistical Methods in Clinical Trials Open access 138 citations 1 result