Sabrina Vorpahl

Sabrina's Fundraiser for Charcot-Marie-Tooth Association

Fundraising for Charcot-Marie-Tooth Association
US$5,677
raised of US$10,000 target
Sabrina Vorpahl's Fundraiser for CMT4C
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

As many of you already know, I struggle with a progressive and debilitating illness called CMT4C. I experience pretty significant nerve pain alongside muscle atrophy and nerve death in my extremities. I received this diagnosis at 15, along with two of my younger sisters, but I had dealt with all symptoms since birth. My life now at 23 looks very different than I ever expected, but this does not have to be my life forever!

For many years we've built a small but strong patient community dedicated to finding and making available a treatment. Dr. Kleopas has recently published incredibly significant findings in his research regarding using a viral vector to edit the exact gene that causes CMT4C. An article discussing his published paper can be found here:

https://journals.lww.com/neurotodayonline/Fulltext/2019/05230/New_Evidence_for_Gene_Replacement_in_a_Mouse_Model.9.aspx

What is needed now is a thorough and specialized Natural History Study, a study of the progression of my illness outside of treatment. Many of the top researchers are already committed to leading this rigorous study, seeing the massive impact that the success of a viral vector in gene editing can have on rare diseases even beyond mine!

Considerable funds are needed to get this Natural History study off the ground. I am determined to find a treatment--if not for me, then for my sisters and their futures. I do not want them to reach 23, disabled from an illness with such a promising treatment available but inaccessible because the funding wasn't there.

Please donate whatever you're able and willing to! You can have a hand is such a remarkable and life-changing step towards a cure!

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$5,677.00
Online donations
US$5,677.00
Offline donations
US$0.00

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