CMTA Donors Pledge $100K for Matching Donations to CMT1A Research

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by Mary Chapman |

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With a $100,000 match pledge to CMT1A research, longtime Charcot-Marie-Tooth Association (CMTA) supporters Seth and Missy Warfield have issued a challenge to other members of the disease community.

The pledge was in celebration of the nonprofit organization earning a prestigious four-star Charity Navigator rating. It’s the highest possible score an organization can achieve for financial health, accountability and transparency. Charity Navigator is the largest and most-utilized evaluator of charities in the United States.

Until Dec. 31, each dollar will be matched up to $100,000. Donations may be made at this website.

The Warfields have a personal interest in the CMTA’s mission to battle Charcot-Marie Tooth and support patients and their families.

“CMT1A has bound three generations of our family together in frustration, awkwardness, understanding, love and fervent hope for a cure soon. We are also bound to all around the globe who daily live with CMT,” the couple said in a statement on the organization’s website.

CMT1A is the most common subtype of CMT1, accounting for about 70% to 80% of CMT1 cases. The disease is caused by a duplication of the PMP22 gene situated on chromosome 17, which provides instructions for making  peripheral myelin protein 22, a critical component of the myelin sheath that surrounds and protects nerves.

It begins with typical CMT symptoms during adolescence — muscle weakness and atrophy of the lower legs, followed by hand weakness and decreased sensations later in life. CMT affects about 3 million individuals globally.

The CMTA’s research centerpiece is its Strategy to Accelerate Research (STAR) program, created to find treatments to slow, stop or reverse CMT progression. Through STAR, which collaborates with investigators globally, the organization is screening new drug candidates and exploring gene therapy approaches to treat CMT1A.

STAR recently approved a project in which scientists will test the distribution of adeno-associated virus 9 (AAV9) in large animal models, and testing new AAV variants for the best way to reach the Schwann cell. The AAV9 vector delivers a healthy gene to affected cells.

Speaking to the need to fund this research, CMTA said “We wholeheartedly thank the Warfield family for their steadfast dedication to stopping CMT by standing behind STAR.”

The Charcot-Marie-Tooth Association supports the development of new CMT therapies, works to improve patients’ quality of life, and searches for a CMT cure.