Bob Paulsen

Quinn's fundraiser for Charcot-Marie-Tooth Association

Fundraising for Charcot-Marie-Tooth Association
US$38,680
raised of US$1,000,000 target
As a Charcot-Marie-Tooth community member your occasion, actions and life events can help the CMTA reach a world without CMT! Create a fundraising page and spread the word. Need help or more information? Contact Mary at mary@cmtausa.org.

Story

Hi. Thanks for spending a few minutes to learn about CMT and what we can do to help advance and deploy a cure.

As you may know, Quinn has had scoliosis since birth. He's also had a number of other challenges with his feet and ankles (which required ankle braces for years), hand strength, balance, feeling, etc. After a decade of searching for a reason, Quinn was only recently diagnosed with CMT (Charcot Marie Tooth) syndrome in July of '23. Since then, we have jumped in to learn as much as we can about CMT, the efforts around it, and what we can do to help.

CMT causes continuous degradation of nerves and muscles over time, in particular to hands and feet. Many with CMT undergo multiple surgeries, have lifelong braces, require wheelchairs and other support systems, and have deformities in their feet and hands. There are about 200k people in the US and ~3M worldwide diagnosed with CMT. Since 1991, there have been over 100 genes identified in causing CMT which have been grouped into a handful of types CMT (CMT type 1, 2, 4, X, etc.), and further by subtypes. Quinn's version is CMT-4C.

The CMT-4C community (Quinn’s type) is small at ~4% of people with CMT. Amazingly, scientists have succeeded in stopping the nervous system degradation in mouse models via a gene therapy for 4C. This is wonderful news for us and others with 4C, but there is still much to be done.

Through great effort and collaboration, we now have aligned some of the best genetic experts in the world to focus on a plan that will drive this gene therapy solutions through the FDA and other logical steps over the next three years. We know the path, now we can accelerate the cure!

Although no one would like to have CMT, we are happy to finally have this diagnosis and learn of the CMTA and of the research progress made by scientists around the world. With a little more financial support, we can fund existing projects and stop the degradation through gene therapy for Quinn and 3 million others across the globe.

Please join us in supporting Quinn and ~200k other Americans with CMT by providing the financial backing that this community needs to reach the goal line and share the cure before things get worse for our young man.

We intend to coordinate a few fundraising events each year. The big one coming up is the Foresee Gala. JOIN US! https://www.cmtausa.org/ways-to-give/4c-gala/ . We'll share more on each event as they come to life as we'd love to see you at any or all that look interesting.

We welcome your support, communication, thoughts, and prayers 24 hours a day. More Life!

About the campaign

As a Charcot-Marie-Tooth community member your occasion, actions and life events can help the CMTA reach a world without CMT! Create a fundraising page and spread the word. Need help or more information? Contact Mary at mary@cmtausa.org.

About the charity

Our mission … to support the development of new drugs to treat CMT, to improve the quality of life for people with CMT, and, ultimately, to find a cure. Our vision … a world without CMT.

Donation summary

Total raised
US$38,680.00
Online donations
US$17,580.00
Offline donations
US$21,100.00

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