Pediatric Rare Diseases: Advancements, Challenges, Future
Received Date: May 02, 2025 / Published Date: May 30, 2025
Abstract
Research in pediatric rare diseases is advancing rapidly. Key areas include gene therapy and CRISPR-based gene editing for
inherited conditions, alongside next-generation sequencing and Artificial Intelligence (AI) for improved diagnosis. Efforts also focus
on regulatory frameworks for orphan drugs, expanding newborn screening, and the crucial role of patient advocacy. Ethical consid
erations in research and the importance of international collaboration and patient registries are also central to accelerating therapeutic
development and patient access.
Citation: Laurent DD (2025) Pediatric Rare Diseases: Advancements, Challenges, Future. NNP 11: 543.
Copyright: 聽漏 2025 Dr. David Laurent This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permitsunrestricted use, distribution and reproduction in any medium, provided the original author and source are credited.
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