Search results for: cmt
CMTA Invests $141,000 to Study Hearing and Balance to Develop Better Clinical Trial Outcome Measures in Charcot-Marie-Tooth Disease
CMTA is supporting a two-year research project examining hearing and balance signaling to strengthen outcome measures in future CMT clinical trials.
Hearing and Balance Biomarkers for Outcome Measure Development in CMT1A
With CMTA support of $141,000, researchers led by Gabriel Corfas, PhD, at the Kresge Hearing...
CMTA Appoints David Stark, MD, and Catherine Illingworth to Board of Directors, Expanding Clinical and Biotech Expertise
CMTA welcomes David Stark, MD, and Catherine Illingworth to its Board of Directors, strengthening clinical insight and gene therapy leadership within the CMT community.
CMTA Addresses the Critical Delivery Challenge Underlying CMT Treatment Development through Four Key Investments
CMTA announced $523,000 in funding for four research projects focused on solving one of the biggest challenges in CMT treatment development: delivering therapies to peripheral nerve cells. By investing in genome-editing transport, blood-nerve barrier crossing, Schwann cell targeting, and nanoparticle systems, CMTA is working to ensure treatments can reach the cells where they are needed.
Schwann Cell-Targeted Peptide-LNPs for Delivery of siRNA Against PMP22 in CMT1A
With combined support totaling $102,968 from CMTA, Shark Tooth Bio, and the National Research Council...
Blood–Brain Barrier-Crossing Conjugates for Treating CMT
CMTA-funded research at Mount Sinai is studying how RNA medicines can be delivered into nerve cells in CMT, overcoming biological barriers that block treatment.
Preliminary Characterization of STEP Platform for Delivery of Genome Editing Therapy for CMT
With CMTA support of $66,000, researchers led by Jiangbing Zhou, PhD, at Yale University are...
CMTA Appoints Wendy Arnone as Board Chair
CMTA has appointed Wendy Arnone as board chair, succeeding Gilles Bouchard, who has led the board since 2016 and will continue serving as a board member. Arnone brings decades of executive healthcare leadership experience and a clinical background as a registered nurse, positioning her to guide CMTA’s continued investment in research and drug development for CMT.
CMTA Center of Excellence at Johns Hopkins All Children’s Hospital
Matias Sebastian Lopez Chacon, MD, is a pediatric neurologist and neuromuscular specialist at Johns Hopkins All...
A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A – (CANADA ONLY)
About this Study The purpose of this study is to characterize the safety, tolerability, and...