Latest Issue – December 2024

In this issue:

  • 2025 Annual Family Conference
  • Conference Sponsorship Opportunities
  • Voice of the Patient Report Published in Human Gene Therapy Journal
  • Prenatal Screening: Billion to One
  • Rare Pediatric Disease Priority Review Voucher Program
  • Giving Tuesday
  • Worldwide Candle Lighting
  • Mission Moment

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FEATURED ARTICLE

Community Connections

GM1 Voice of the Patient Report Published in the Human GeneTherapy Journal

NTSAD is proud to share that our Voice of the Patient report from the 2024 Externally-Led Patient-Focused Drug Development meeting with the FDA and other key stakeholders was recently published in the Human Gene Therapy Journal. Spreading awareness about the urgent need for effective treatments and informing regulators about living with the ultrarare diseases we represent is an essential role we play as patient advocates. Thank you to every member of the NTSAD community whose voice was captured in this report.

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Community Connections

Get the latest news from NTSAD about our Community, the research that provides hope, and what is happening in the world of rare disease.