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Gene Therapies for Rare Diseases Twice as Likely to Win FDA Nod, Study Finds

Summary by Managed Healthcare Executive
None of the cell and gene therapies that reached the final regulatory submission stage were rejected by the FDA.
DisclaimerThis story is only covered by news sources that have yet to be evaluated by the independent media monitoring agencies we use to assess the quality and reliability of news outlets on our platform. Learn more here.

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pharmtech.compharmtech.com

INTERPHEX 2025: Challenges in Ultra-Rare Disease Treatment Development

The Pharmaceutical Technology® Group sat down with Joe Katakowski, director of Research at the RTW Foundation about the challenges involved in the development of treatments for ultra-rare diseases.

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Managed Healthcare ExecutiveManaged Healthcare Executive

Gene Therapies for Rare Diseases Twice as Likely to Win FDA Nod, Study Finds

None of the cell and gene therapies that reached the final regulatory submission stage were rejected by the FDA.

Read Full Article
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Managed Healthcare Executive broke the news in on Thursday, April 3, 2025.
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