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September 5, 2025International Journal of Stem CellsOpen Access

Advances in Cell and Gene Therapy for Rare Disease Treatment

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Authors

HBHyun Jin BaekYNYoojun NamSungkyunkwan UniversityYRYeri Alice RimSt. Mary's Hospital

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Overview

Review reveals advancements in cell and gene therapies for rare diseases, highlighting orphan drug development strategies.

Key Points

  • The orphan drug market is expanding rapidly, indicating a promising growth in rare disease treatments.
  • Cell therapies and gene therapies are at the forefront of advanced therapy medicinal products in orphan drug development.
  • The review considers strategic approaches that enhance the competitiveness of the orphan drug industry using advanced therapies.
  • Advancements since the 1990s showcase increasing investment and support for rare disease research and development.

Cite This Study

Baek et al. (2025) studied this question.

synapsesocial.com/papers/68bb46c96d6d5674bccff137https://doi.org/10.15283/ijsc25028
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