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Contribution of Dr. Mark Batshaw in Field of Pediatrics

Goswami Sanjana Sanjay, Kasumbiwal Ajay H, Male Rohit H, Narwate Tukaram

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Pediatric Education and Research 13((Special Supplement -I)):p 133-137, July-December 2025. | DOI: 10.21088/per.2321.1644.13225.18

How Cite This Article:

Kasumbiwal Ajay H., Male Rohit H., Tukaram Narwate B., et al. Contribution of Dr. Mark Batshaw in Field of Pediatrics. Pediatr. Edu. Res. 2025; 13(2): 133-137.

Timeline

Received : November 24, 2025         Accepted : December 25, 2025          Published : December 30, 2025

Abstract

Dr. Mark L. Batshaw is a distinguished pediatrician, researcher, and educator whose work has transformed the landscape of developmental medicine and rare disease research. Renowned for his pioneering contributions to the understanding and treatment of urea cycle disorders, Dr. Batshaw helped develop innovative therapies that have significantly improved survival and quality of life for affected children. His leadership at Children’s National Hospital and his role in the NIHfunded Rare Disease Clinical Research Network have shaped collaborative research models now used worldwide. Beyond his scientific achievements, Dr. Batshaw is widely respected for his authorship of Children with Disabilities, a foundational textbook that has guided generations of clinicians in caring for children with developmental and genetic conditions. His personal experiences with learning challenges cultivated a lifelong commitment to compassion, advocacy, and patientcentered care. Through his research, mentorship, and dedication to advancing pediatric science, Dr. Batshaw has left an enduring legacy that continues to influence modern clinical practice and inspire future healthcare professionals. Key Message: A pioneer in developmental pediatrics, Dr. Mark L. Batshaw revolutionized the understanding and treatment of childhood genetic and metabolic disorders.


References

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  • 2.   Brusilow S.W., Maestri N.E. Urea cycle disorders: diagnosis, pathophysiology, and therapy. Adv Pediatr. 1996; 43: 127-70.
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  • 4.   Batshaw M.L., Walser M., Brusilow S.W. Alternative pathway therapy for urea cycle disorders. N Engl J Med. 1982; 306(23): 1387-92.
  • 5.   Batshaw M.L., Tuchman M., Summar M., Seminara J. A longitudinal study of urea cycle disorders. Mol Genet Metab. 2014; 113(1-2): 127-30.
  • 6.   Lee B., Goss J., Barshop B., Yu H., Zahl T., Bartz D., et al. Chronic treatment of urea cycle disorders with glycerol phenylbutyrate. Mol Genet Metab. 2010; 100(3): 229-34.
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  • 8.   Wilson J.M. Lessons learned from the Gelsinger case: ethical considerations in human gene therapy. Hum Gene Ther. 2009; 20(9): 848-56.
  • 9.   Wang L., Wang H., Bell P., McCarter R.J., He J., Calcedo R., et al. Systemic AAV8mediated gene therapy for ornithine transcarbamylase deficiency. Mol Ther. 2010; 18(4): 7 13-21.
  • 10.   Cunningham S.C., Putt W., Arnell H., Taylor M, Alexander I.E. Gene therapy for urea cycle defects: current progress and future prospects. J Inherit Metab Dis. 2020; 43(1): 41-55.
  • 11.   Yang Y., Wang L., Bell P., McMenamin D., He Z., White J., et al. A dual-vector CRISPR/Cas9 system for OTC gene correction in newborn mice. Nat Commun. 2016; 7: 11900.
  • 12.   Batshaw M.L., Roizen N.J., Lotrecchiano G., editors. Children with Disabilities. 8th ed. Baltimore: Paul H. Brookes Publishing; 2019.
  • 13.   Batshaw M.L., editor. The Child with Special Needs: Developmental and Behavioral Pediatrics. Baltimore: Williams & Wilkins; 1993.
  • 14.   Vieira K.A., Rosenberg L.A., Dias T., McCarter R., Batshaw M.L. Personal reflections on learning disabilities in medicine. Pediatrics. 2014; 133(5): 789-92.
  • 15.   DeBenedictis R., Kellogg M., Batshaw M.L. The evolution of developmental pediatrics: contributions of the Kennedy Krieger Institute. Dev Med Child Neurol. 2012; 54(3): 198-204.

Data Sharing Statement

There are no additional data available. All raw data and code are available upon request.

Funding

This research received no funding.

Author Contributions

All authors contributed significantly to the work and approve its publication.

Ethics Declaration

This article does not involve any human or animal subjects, and therefore does not require ethics approval.

Acknowledgements

We would like to express our gratitude to the patients, their families, and all those who have contributed to this study.

Conflicts of Interest

The authors report no conflicts of interest in this work.


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Cite this article

Kasumbiwal Ajay H., Male Rohit H., Tukaram Narwate B., et al. Contribution of Dr. Mark Batshaw in Field of Pediatrics. Pediatr. Edu. Res. 2025; 13(2): 133-137.


Licence:

Attribution-Non-commercial 4.0 International (CC BY-NC 4.0)

This license enables reusers to distribute, remix, adapt, and build upon the material in any medium or format for noncommercial purposes only, and only so long as attribution is given to the creator


Received Accepted Published
November 24, 2025 December 25, 2025 December 30, 2025

DOI: 10.21088/per.2321.1644.13225.18

Keywords

Gene therapyMark BatshawLearning disabilitiesAAV vector

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Received November 24, 2025
Accepted December 25, 2025
Published December 30, 2025

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Attribution-Non-commercial 4.0 International (CC BY-NC 4.0)

This license enables reusers to distribute, remix, adapt, and build upon the material in any medium or format for noncommercial purposes only, and only so long as attribution is given to the creator


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