Susan Ruediger on Pursuing Gene Therapies for CMT

“Our job at the Research Foundation is to bring CMT to the forefront of everybody's mind, and then to bring a little bit of funding along with it to motivate people to get involved. And without that effort, and without that focus, if not us, then who? And if not now, then when? So that's the perspective that patients like me can bring is that sense of urgency and the sense of organization.”

As more companies explore gene and cell therapies for rare diseases like Charcot-Marie-Tooth (CMT) disease and others, the importance of collaboration between industry, academics, and patient advocacy groups has become more and more apparent to successfully develop and evaluate these therapies.

The nonprofit company CMT Research Foundation is working to help researchers find treatments for CMT. Susan Ruediger, the company’s founder and chief mission officer, who herself has CMT, recently participated in a discussion entitled “Partnering for Progress”, on this topic during the 2022 BIO International Convention, held June 13-16 in San Diego, California.

CGTLive spoke with Ruediger to learn more about the CMTRF and its mission. She also discussed how having CMT herself has driven her to found the company and the unique perspectives patients can bring to therapy research and development.

REFERENCE

Schmidt H, Ruediger S, et al. Partnering for progress: Collaborating with patient organizations to advance therapy development and maximize patient benefit. Presented at: 2022 BIO International Convention, June 13-16, San Diego, CA.