A message from Dynacure's Patient Advocacy Lead, Lori Gorski, to our MTM-CNM Community:
My name is Lori Gorski and I oversee Patient Advocacy for Dynacure. Our company has just begun a Phase 1/2 study of an investigational therapy for the treatment of Myotubular and Centronuclear Myopathies. We are looking forward to learning more about MTM and CNM through these early clinical studies, and through our engagement with you. Dynacure is headquartered in Strasburg, France and our study is currently underway in Europe. If successful in this first study, we will look to pursue additional clinical trials that include participants in United States. As our plans progress, we are eager to learn more about the patients, families and caregivers here in the US.
Here's my ask: I am looking for about six patients across the country who would agree to share their stories with me. I am most interested in learning about your path to diagnosis, how MTM/CNM affects your life and about your ambitions and hopes for the future. My goal is to eventually share these stories with the entire Dynacure team, and externally on social media and on our Website as a way to increase general awareness and understanding of this disease. I know just six stories cannot entirely capture the unique journeys and experiences within CNM/MTM families, and I am eager to continue our learning in other ways in the months ahead.
If you'd be willing to share your story, I ask that you send an email directly to me at firstname.lastname@example.org and I will share more details about this project with you. We can set up a time to speak by phone at your convenience for a one-hour interview. After we speak, I will share a draft of your story with you to be sure you are comfortable with what we captured. In a perfect world, I'd love to come and meet with you in person, but those plans must remain on hold for now due to COVID.
Thank you for the opportunity to learn more about CNM and MTM. I am grateful to each of you, and to the devoted leaders of the CNM/MTM patient organizations for your collaboration and guidance.
Lori Gorski, Patient Advocacy