Monique Manzella and JP Ross

Let's help Keenan's family raise funds for a CMT 4c Cure!

Fundraising for Charcot-Marie-Tooth Association
US$30,176
raised of US$40,000 target
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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

Our 15 year old son, Keenan, has recently been diagnosed with a rare genetic neurological disease, Charcot Marie Tooth, type 4c. This is a progressive disease affecting the protective myelin coating of peripheral nerves, so his mobility and balance are at risk for worsening as he gets older. CMT degenerates the nerves and muscles of the feet, hands and extremities. But peripheral nerves are all throughout the body and this degeneration can cause many other symptoms, such as eye conditions, hearing loss, fatigue, and joint and nerve pain. Keenan has two amazing neurologists Dr. Fay of UCSF and Dr. Kleopa in Cyprus.

The good news is that hope is on the horizon for a cure! The research is promising! The first and essential step of identifying the specific gene and mutation is done. Second, there has been a successful mouse study using gene replacement therapy, successfully resulting in STOPPING the progression of the disease and even gaining back some function.

The next step is a natural history study that measures disease progression in CMT4c patients over the course of 2-3 years. This study has recently been designed, written and submitted for approval by two of the leading CMT neurologists in the world, Dr. Kleopa of Cyprus and Dr. Hermann of the US. We are currently fundraising for this part of the research. Once the natural history study is completed, we should be very close to human clinical trials! CMT 4c patients are organized and ready to participate!

Once gene therapy is available for CMT 4c, these patients would require only one injection to stop this disease from progressing. Gene therapy research and treatment is a promising cure for many other genetic diseases. By contributing to the cure of CMT 4c, you will help the gene therapy model of treatment be used to cure other genetic diseases as well.

We are part of a small but mighty CMT 4c patient community working with the CMTA to raise funds for a cure for CMT 4c.

Keenan is an awesome kid. He is earning all A's in school, and especially enjoys physics, robotics, and computer science. He loves swimming, hanging out with his family and spending time with his best friend. He's always authentically himself and is super funny.

We've set our fundraising goal at $25,000 but this research into clinical trials will likely cost far more. This will be a great start though!

Please help us move CMT 4c research forward and to reach a cure! It could be the difference of Keenan being able to walk independently his whole 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$30,175.57
Online donations
US$30,175.57
Offline donations
US$0.00

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