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Transformative effect of a Humanitarian Program for individuals affected by rare diseases: building support systems and creating local expertise |
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| | I. C. Verma, A. El-Beshlawy, A. Tylki-Szymanska, A. Martins, Y.-L. Duan, T. Collin-Histed, M. Schoneveld van der Linde, R. Mansour, V. C. Dung, Pramod K. Mistry | | | Orphanet Journal of Rare Diseases. 2022; 17(1) | | | [Pubmed] [Google Scholar] [DOI] | | | 2 |
Advances in orphan drug development: Time to change the status and stereotype |
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| | Sayanta Thakur | | | Perspectives in Clinical Research. 2022; 13(3): 123 | | | [Pubmed] [Google Scholar] [DOI] | | | 3 |
Challenges in Chronic Genetic Disorders: Lessons From the COVID-19 Pandemic |
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| | Swasti Pal, Sameer Bhatia, Sunita Bijarnia-Mahay, Ishwar C Verma, Ratna Dua Puri | | | Indian Pediatrics. 2021; 58(4): 391 | | | [Pubmed] [Google Scholar] [DOI] | | | 4 |
Treatment for Lysosomal Storage Disorders |
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| | Jayesh Sheth, Aadhira Nair | | | Current Pharmaceutical Design. 2020; 26(40): 5110 | | | [Pubmed] [Google Scholar] [DOI] | |
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