CMTA Announces $150,000 Research Challenge Fundraiser for CMT Disease

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by Mary Chapman |

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Gene therapy

 To help it reach its fundraising goal by year’s end, the Charcot-Marie-Tooth Association (CMTA) has issued its 35th Anniversary $150,000 Research Challenge.

Donations will be matched dollar for dollar through Dec. 31, 2018. Fundraising totals are continually updated on the CMTA website.

 Funds will go toward investigations led by the organization’s Strategy to Accelerate Research (STAR) program and 27 scientific advisers. STAR has five key elements: Assays, (tests), animal models, stem cells, partners, and clinical trials. This year, researchers are working on some 18 projects involving Charcot-Marie-Tooth. 

 Over the last two years, CMTA has increased its research partnerships from two to 20 with pharmaceutical, biotechnology and research service companies. Since its inception, the organization has steered $8 million to research.

 Go to the website to make a donation.

Initially called the National Foundation for Peroneal Muscular Atrophy, the nonprofit organization was established 35 years ago after a meeting in New York of CMT families and physicians, according to the website’s timeline. 

 In 1995, the CMTA began awarding fellowship grants to CMT researchers; in 2001, in conjunction with Wayne State University, a patient database was created for North America.

 Owing to generous contributions by two CMTA families, the STAR program was created in 2008. Two years later, the organization brought together globally recognized CMT specialists for a CMT Type 2 research symposium. In 2012, CMTA established 11 Centers of Excellence to help patients get the best possible evaluation and care.

  In 2014, the organization added CMA types 1X, 1B, 2A, 2E, and 4 to its research platform. Last year, in collaboration with the pharmaceutical company Ionis, the CMTA announced a major breakthrough in CMT1A gene-targeting research.

 A chief goal now is accelerating into clinical trials breakthroughs in gene and broad-based therapies for all types of CMT.

 Affecting about one in 2,500 people and 3 million people worldwide, Charcot-Marie-Tooth is a slowly progressive disease of the peripheral nerves, which control muscles. Although there is no drug treatment for CMT, physical therapy and moderate activity can help maintain muscle strength, endurance and flexibility. Occupational therapy and adaptive devices can assist in daily living. A CMTA Guide to Physical and Occupational Therapy can be downloaded here.