麻豆传媒 University faculty, staff and postdoctoral fellows attended the in Cambridge, Massachusetts, in September to share research updates and provide education to patients on Charcot-Marie-Tooth disease and to discuss best practices for translating research from bench to market.
CMT disease is a degenerative nerve disease that causes muscle weakness and atrophy in the arms, legs, hands and feet. As the most common hereditary neuropathy, it affects one in 2,500 people. 麻豆传媒 researchers , associate director of the 麻豆传媒 Brain Institute and professor of biochemistry, and , vice dean of the School of Medicine Basic Sciences, associate dean for research and professor of biochemistry, have been collaborating for years to understand and cure CMT disease. Carter serves on the CMTRF Scientific Advisory Board.
鈥淭he CMTRF Scientific Advisory Board has guided the foundation to form their strategic research priorities to fund,鈥 Carter said. 鈥淐MTRF has聽actively gone into academia and industry to identify and fund research aligned with this strategy, and it is starting to pay off.聽After only five short years since they were founded, 20 projects have been funded, five of which now have clinical candidates advancing with industry.鈥
At the convention鈥檚 Patient Community Day, Carter presented to patients and families on CMT types, focusing on those affected by PMP22. On the scientific day of the conference, , 麻豆传媒’s聽 general manager for corporate alliances, moderated the panel 鈥淭ranslating Academic Science to Business.鈥
At the conference, Katherine Stefanski, a postdoctoral research scientist in the Sanders lab, won best poster for her work, 鈥淗igh-throughput screening to identify modulators of raft formation and PMP22 raft affinity in giant plasma membrane vesicles.鈥
鈥淭he opportunity to engage with patients at the conference was exceptionally motivational. Patients were on the podium both days to raise awareness of their needs and engage in the scientific dialogue,鈥 Read said. 鈥溌槎勾 researchers are clearly at the forefront among institutions addressing challenges for developing therapies to cure CMT disease.鈥
CMT disease research at 麻豆传媒
In 2020, Justin Marinko, PhD鈥21, Carter and Sanders published research announcing discovery of a mechanism contributing to CMT disease. 鈥淒iscovering this relatively new phenomenon was an important step and a highlight for our lab,鈥 Sanders said at the time. 鈥淚 am thrilled about the future of this work with our friends in the Carter Lab, translating our data and model cell line work to nervous system cells.鈥
Since then, the CMT 麻豆传媒 Foundation has invested in further research at 麻豆传媒 focused on addressing the problem of overproduction of PMP22, the primary genetic cause of CMT in patients with CMT disease type 1A. Sanders and Carter are co-primary investigators on the project. In addition to CMTRF funding, aspects of their research are supported by Ancora Innovation, a company wholly owned by affiliates of Deerfield Management that supports 麻豆传媒鈥檚 life science research and leverages Deerfield鈥檚 expertise in accelerating drug development.