Most people in the GNEM community ask questions such as the following: (1) Why is there not a cure yet? (2) What is the NDF doing to promote a cure? (3) When will the next ManNAc trial begin? (4) When will gene therapy begin? At the NDF Fifth Annual Symposium these questions and more questions were answered. There are over 7,000 rare diseases for which only 5% have approved therapies. Therefore we are quite fortunate that our disease (GNEM) has been attracting more doctors and researchers to collaborate with organizations like the NDF to find an effective cure.
The NDF has been asking: "Why is there not a cure yet?" It has been pursuing an effective therapy since its inception in 2006. This is the reason, the NDF has been having yearly Symposiums to connect the brightest scientific minds involved in GNEM research with GNEM patients face-to-face. The reason NDF exists is to get a cure for GNEM patients, and it has never faltered from its mission. A CURE and this pursuit continues to be its modus operandi (MO).
The focus this year consisted of a continuation from the last year's Symposium that brought researchers and patients together to continue exploring every avenue for a possible cure. Each year the researchers present their current findings and research. This Symposium was attended by patients from throughout the world specifically the Ivory Coast, Italy, Israel, Germany, India, Korea, Nepal, Canada, and the United States. Many patients received travel stipends, accommodations, and meals during this time.
You should be aware of the barriers in your home/work place that affect your activity. You should ensure that you have safe mobility and functioning in your environment.
The investigators are Dr. Amato (Harvard) and Dr. Nuria Carrillo (NIH). Pharma Company partner for the trial is- Leadiant Biosciences
Patient session (Video Link)
After all the sessions of the first day, there was a reception/dinner accompanied with live music. At this dinner the NDF enlisted four more Certified Patient Advocates from Germany, India, the Ivory Coast, and Israel.
Besides the many events of the Symposium, we were able to see through the eyes of Saskia, a GNEM patient from Germany a video log of her trip to Peru. Here is a link to her Jungle Expedition:https://www.youtube.com/watch?v=QA4x2vSkUjQ&feature=youtu.be
Also, Dr. Sepideh Tabibian presented Holist Approach for Coping with GNEM. Here is a link to her presentation: https://www.facebook.com/NDF.HIBM/videos/vl.2159531020989886/500981303706758/?type=1
Upcoming Events within our GNEM community:
1. WWGM is participating in Airtel Delhi Half Marathon on October, 21, to build awareness around GNEM and to raise funds towards research and treatment
For more, please visit: https://www.facebook.com/SupportGNEMyopathy/
2. NDF Gala for the Arts: Join the NDF on November 11, 2018 to celebrate a unique evening or art and music.
The NDF is looking to raise funds to support a $2.5 million challenge grant provided by a private foundation. The funds raised will support our efforts toward therapies for GNE Myopathy. For more, please visit:http://curehibm.org/