Research News and Updates

Read the latest from the CMT Research Foundation on drug development and clinical advances.

2022 at CMTRF

by Chelsea Layton | Jan 9, 2023 | CMT Research Updates, CMTRF Funded Research, News

Dear Friends of CMTRF, In preparing for another busy year at CMTRF, I took some time recently to look back at the accomplishments of 2022. I am immensely proud of what our team has achieved (summarized below) and I believe it underscores how CMTRF adds significant...

Muscular Dystrophy Association and CMT Research Foundation Announce Joint Research Study Grant

by Chelsea Layton | Dec 14, 2022 | CMTRF Funded Research, Press Releases

(New York) December 14, 2022 The Muscular Dystrophy Association (MDA) and CMT Research Foundation (CMTRF) announce joint funding for a study by Alessandra Bolino, PhD, at San Raffaele Hospital (Ospedale San Raffaele), Italy, on restoring membrane trafficking in...

Dr. Arthur Suckow, DTx Pharma Inc. – FALCON

by Chelsea Layton | Nov 28, 2022 | CMTRF Funded Research, Drug Development, Stories

One of the first research teams the CMT Research Foundation (CMTRF) funded was DTx Pharma, Inc., and the project focused on delivery of genetic medicine to the myelinating Schwann cells of the peripheral nerves - a major scientific challenge to effectively treating...

CMT Research Foundation partners with Samsara Therapeutics to develop a novel class of drugs in CMT1A

by Chelsea Layton | Oct 13, 2022 | About CMT, CMT Research Updates, CMTRF Funded Research, Research news

By: Keith Fargo, PhD, Chief Science Officer The CMT Research Foundation is pleased to announce a new project with Samsara Therapeutics, a US/UK-based biotech company that is developing a novel class of drugs for CMT1A. CMT1A is caused by duplication of a stretch of...

CMT Research Foundation and Samsara Therapeutics Partner on a Novel Therapeutic Approach for CMT1A with the Potential to Reach Clinical Trials

by Chelsea Layton | Oct 12, 2022 | CMTRF Funded Research, Press Releases, Research news

ATLANTA (October 13, 2022) The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease*, has invested in Samsara Therapeutics Inc., a biotech company focusing on discovering and developing...

CMT Research Foundation Awards $152,524 to ORYZON to test novel HDAC6 inhibitors in CMT1A

by Chelsea Layton | Jul 26, 2022 | CMTRF Funded Research, Research news

By: Keith Fargo, PhD, Chief Science Officer New project will examine impact of two selective and potent drug candidates recently discovered by ORYZON The CMT Research Foundation is delighted to announce a new project with ORYZON, a clinical stage pharmaceutical...

ORYZON collaborates with the CMT Research Foundation in the US

by Chelsea Layton | Jul 26, 2022 | CMTRF Funded Research, Press Releases, Research news

CMTRF to provide funding for in vivo efficacy tests of Oryzon’s HDAC6 inhibitors in Charcot-Marie-Tooth preclinical model MADRID, SPAIN and Atlanta, July 26, 2022 Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company...

The CMT Research Foundation Invests Over $500,000 to Find a Single Gene Therapy for Possibly Every Form of CMT1B

by Chelsea Layton | Jul 18, 2022 | CMT Research Updates, CMTRF Funded Research, Research news

By: Keith Fargo, PhD, Chief Science Officer CMT1B is one of the most common forms of CMT. In CMT1B, the part of the nervous system that is dysfunctional is the myelin sheath of the peripheral nerves. Created by cells called Schwann cells, myelin wraps around the parts...

Project Testing PIKfyve Inhibitor in CMT4B1 Clears First Hurdles

by Chelsea Layton | Jul 11, 2022 | About CMT, CMT Research Updates, CMTRF Funded Research, Research news

By: Keith Fargo, PhD, Chief Scientific Officer We are happy to report that a CMTRF-funded project designed to test a PIKfyve inhibitor in CMT4B1, a particularly devastating form of the disease, has passed the first stage and is moving on to the next stage, a...