Page 10 - 2021 Spring CMTA Report - Special Research Edition

MARKING 38 YEARS OF GROWTH

ACCELERATING RESEARCH. EMPOWERING PATIENTS.

Dr. Shapiro

2020
Gene therapy projects in progress for 1A, 1X, 2A, 2E, 2F, 4C, and 4A with world-class labs and four biotech partners.
SORD discovered. Working with Pharnext on biomarkers for its 2021 1A trial.
2019
Passage Bio commits to CMT2A gene therapy CRISPR project with top lab at UC (associated with 2020
Nobel prize laureate), CMTA partner InFlectis BioScience completes Phase 1 clinical trial for Sephin (1A and
1B potential). Renewed support for the INC.
2018
Gene therapy summit. New gene therapy projects and SAB members. Multi-year, multi-partner initiative to test
drugs developed to slow axon degeneration in many CMT models.
2017
In collaboration with Ionis, the CMTA announces a major breakthrough in CMT1A research.
2016
The CMTA establishes Camp Footprint - the first camp in the US for children living with CMT.
2013
Dr. John Svaren, from the University of Wisconsin, creates state-of-the-art cell lines employing genome editing technology,
while other scientists in the CMTA network develop and utilize human stem cells in CMT research.
2012
The CMTA establishes 11 Centers of Excellence to help ensure CMT patients receive the best possible evaluation and
care, and their information is collected for possible recruitment into clinical trials.
2011
CMTA board member Elizabeth Ouellette organizes the first CMT Awareness Week. Her eŠort sparks an international
movement and becomes an annual month-long celebration to spread awareness about CMT.
2010
The CMTA brings together globally recognized CMT specialists for a CMT Type 2 research symposium in San Diego, CA.
2009
Less than one year after STAR is set into motion, the first CMT1A cell line is grown. CMT1A drug discovery begins using
high throughput screening at the National Institutes of Health.
2008
In a vote of confidence for the CMTA’s new research initiative, two CMTA families make large contributions and kick start
fundraising campaigns to support the Strategy to Accelerate Research (STAR).
2006
Joining forces with the MDA, the CMTA funds the first-ever ascorbic acid clinical trials.
2003
CMT advocate and volunteer Bob Budde becomes the liaison for CMTA support groups.
2001
In conjunction with Wayne State University, Dr. Michael Shy and the CMTA establish the North American CMT Database.
1995
The CMTA Board of Directors begins awarding $35,000 fellowship grants to CMT researchers.
1991
Dr. James Lupski, MD, PhD, identifies the gene that causes CMT1A.
1983
The CMTA’s predecessor, the National Foundation for Peroneal Muscular Atrophy, meets for the first time.

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