Each September we join with other organizations to build awareness for Charcot-Marie-Tooth Disease. As you know, CMT is little-known and poorly understood outside of the patient community and we celebrate each and every effort to educate the medical community, the pharma and research communities and the world population in general.
The community that makes up the CMT Research Foundation is focused on action in addition to awareness during this month. Because the CMT community took such powerful action in September 2020 by raising more than $20,000, the CMT Research Foundation was able to move quickly on several urgent projects.
Here is a look at 5 new projects we brought online:
In October 2020 we funded two new projects. One, that focuses on CMT1B and tests the role of inflammation in the disease, is run by Dr. Rudolf Martini at University of Wurzburg, a widely respected CMT expert scientist. He reports making great progress. The other project run by Drs. Chris Lorson and Michael Garcia at University of Missouri is building a precise gene therapy for CMT2E. If effective, it will address all mutations of CMT2E.
In January 2021 we funded Dr. James Dahlman at Georgia Tech to effectively deliver gene therapies into the peripheral nerves, a major challenge in CMT treatment. Dr. Dalman also reports making great headway on his project.
In June 2021 we funded Dr. Charles Abrams to investigate how inosine may help relieve the symptoms in CMT1X. As a world leader in CMT1X, we are confident that this project could be significant.
In June 2021 we introduced Dr. Alessandra Bolino to AcuraStem to investigate how one of AcuraStem’s drugs could be transformational for people living with CMT4B1. We funded the project, and all parties are excited about the promise of this project.
Currently we are sourcing many more gene therapy and small molecule projects for different types of CMT. We will share news about those projects once they’re approved.
The CMT Research Foundation now has projects in all of the predominant forms of CMT, along with several of the rarer types. We are hopeful that any progress against any type of CMT may translate into therapies for other types of the condition.
Two of our early-funds projects have made significant progress: DTx and Armatus.
Both have advanced enough to earn outside investor support and are now preparing the therapies for potential clinical trials, starting as early as 2023.
We’re planning a life-changing scientific convention
To identify more barriers to drug development and develop strategies to overcome them, this September, CMTRF is hosting the first global conference of patients, scientist, government, pharma, academia and other researchers involved in trying to solve CMT. We hope you will consider attending in person or joining the proceedings that will be streamed online. It will be a great way to culminate CMT Action Month!
We get things done with the donations you give to us. And we have built a track record of spending donations wisely, efficiently and on just on research. We are focused solely on finding a cure for CMT and funding translational projects that continue to show positive results.
The promise and progress can only be sustained – and grow – if you continue to provide financial support through donations. There has never been a time in the history of this disease when treatments – or even a cure – are so close. Let’s all pull together to make our dream of ending CMT in our lifetimes a reality.
We are now kicking off September Action Month 2021, with a goal of raising $25,000. Every donation you make will be MATCHED by a generous donor to the Foundation during this month.
You can either give directly, or start a personal fundraiser. Both are easy and very valuable actions you can take right now to advance CMT research.
CREATE YOUR PERSONAL FUNDRAISER AND SHARE WITH FRIENDS AND FAMILY
hello there my name is mick gaffney i live in australia and i have cmt im trying to find out if i can get some research done on pxt 3003 here in australia but nobody here seems too know much about this drug is there anyway you could help me here, with this problem i would love to come too america and get some help with my cmt is there anyway you could me with my cmt, it is driving me crazy as you could understand.
Cheers Mick GAFFNEY