Welcome to the Gorlin Syndrome Alliance Natural History Study (NHS)

The NHS is a platform for individuals with Gorlin syndrome to share their experiences, treatments, symptoms, milestones, and daily challenges. By collecting this valuable information, the Gorlin Syndrome Alliance Natural History Study (NHS) helps the community and researchers to:

• Gain a deeper understanding of the full spectrum of Gorlin syndrome
• Identify trends that reveal new insights and areas for further study
• Foster collaborations with academic institutions and pharmaceutical companies
• Guide the development of care guidelines and best practices
• Support clinical trials by connecting participants with relevant research studies
• Keep personal medical information organized and secure in one place
• Accelerate progress toward new treatments and solutions

Hosted by the National Organization for Rare Disorders (NORD), the NHS was created according to current best practices for patient natural history studies. Its surveys, protocols, and data use policies have been reviewed and approved by an independent ethics committee (Institutional Review Board, IRB), and all data is collected only with participants’ informed consent.

This ensures the information can be responsibly used by academic, government, and industry researchers (e.g., NIH, FDA) and published in scientific journals. The process for linking participants to clinical studies has also been reviewed and IRB-approved, supporting safe and ethical research participation. All natural history study data is stored securely and maintained in compliance with patient privacy regulations. All information is de-identified to protect privacy, and every contribution adds to a growing, powerful resource for both patients and researchers.

Video links:

• What is a Registry?
• Janet Woodcock, Former Director CDER FDA on NORD Registry Program