| 1 |
Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades |
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| Venkateswaran Vellaichamy Swaminathan, Satishkumar Meena, Harika Varla, Rumesh Chandar, Indira Jayakumar, Balasubramaniam Ramakrishnan, Ramya Uppuluri, Revathi Raj |
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| Indian Pediatrics. 2022; 59(9): 699 |
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| [Pubmed] [Google Scholar] [DOI] |
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| 2 |
Lysosomal storage disorders: From biology to the clinic with reference to India |
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| Jayesh Sheth, Aadhira Nair, Babban Jee |
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| The Lancet Regional Health - Southeast Asia. 2022; : 100108 |
|
| [Pubmed] [Google Scholar] [DOI] |
|
| 3 |
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] |
|
| 4 |
Advances in orphan drug development: Time to change the status and stereotype |
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| Sayanta Thakur |
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| Perspectives in Clinical Research. 2022; 13(3): 123 |
|
| [Pubmed] [Google Scholar] [DOI] |
|
| 5 |
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 |
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| Indian Pediatrics. 2021; 58(4): 391 |
|
| [Pubmed] [Google Scholar] [DOI] |
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| 6 |
Treatment for Lysosomal Storage Disorders |
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| Jayesh Sheth, Aadhira Nair |
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| Current Pharmaceutical Design. 2020; 26(40): 5110 |
|
| [Pubmed] [Google Scholar] [DOI] |
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