RARE Drug Development Symposium: Cell Models in Rare Disease Research
**Day 1: **Optimizing the use of cell models in rare disease research can sound daunting. Dr. Deborah Requesens and Herve Tiriac discuss how cell models can be used to set a strong foundation for your research and encourage diversity in clinical trials. **Day 2: ** The value of cell models lies in setting a good fou…
RARE Webinar: Understanding Gene Therapy
[Recorded webinar][1]/[Download slides ][2] Scientific discovery is advancing rapidly, and new frontiers in gene therapy may yield promise for new rare disease treatments. You may have heard the term gene therapy and even know a little bit about the science, but this webinar will cover all the basics and walk through…
Cell and Gene Therapy for Rare Disease Clinical Trials FAQ
**Cell and gene therapies hold great promise for some rare diseases. After a rare disease is discovered, research continues to better understand the disease and explore possible treatment options - which can include cell and gene therapies. Clinical trials are an import- ant step in helping scientists understand how w…
Cell and Gene Medicine
Rare disease patients say their biggest concern about exploring a specific gene medicine is being able to make an informed decision about their care. Cell and gene medicine science has moved at an unprecedented pace in the past few years, and researchers have worked tirelessly to harness new tools and technologies for…
