Lori Gorski has recently joined the team at Dynacure, leading Patient Advocacy. Below she introduces herself and writes about the work currently being undertaken by Dynacure.
My name is Lori Gorski and I am one of the newest members of Dynacure. In my role leading Patient Advocacy, I am your official connection to our team. I will be responsible for keeping you updated on our research efforts and for making sure your voice and perspectives are front and center within Dynacure.
As we near the end of 2020, I want to acknowledge the challenges of this year. It is not lost on anyone at Dynacure that this is an extraordinarily burdensome year for the rare disease community, and particularly intense for the myotubular and centronuclear myopathy community. Together with you, we are hopeful for brighter days to come.
I would like to share a few updates with you:
- This year, we have started to dose the first patients with our investigational product candidate, DYN101, in a Phase 1/2 study called UNITE-CNM. The goal of the study is to learn about the safety and tolerability of DYN101 in patients age 16 and above, with X-linked or autosomal dominant CNM (or in patients with MTM1 or DNM2 mutation). UNITE-CNM is currently running in the following European countries: Belgium, Denmark, France, Germany, United Kingdom and The Netherlands. More information can be found on the EU Clinical Trials Register.
- We are developing case studies about patients and their families, beginning in the U.S. and expanding to include Europe as well. Our goal is to share these stories externally on social media and on our website to increase disease awareness and understanding. If you are interested and able to share your story, please reach out to me at firstname.lastname@example.org.
- We have posted new organisation guidelines about how as a company we work with patient organizations and community advocates. I hope you find it helpful. You can reach us at email@example.com if you have questions or feedback.
Finally, on behalf of everyone at Dynacure, please accept our heartfelt thanks and appreciation for your input and support as we begin our early studies and work to advance our understanding of myotubular and centronuclear myopathies. We aspire to be good partners and students of your community, and please know that you inspire each of us in our work each day.