Small Molecule Therapies for CMT
Small molecule therapies typically are developed from simple building blocks or are derived from natural sources. With their small size, they are easily able to pass through the cell membrane and access targets inside the cell.
Small molecule drugs are designed to modify a disease process via regulation of a biological target such as an enzyme, channel or receptor. They make up the majority of today’s drug treatments and may be used alone or as individual elements in combination therapies.
CMT Research Foundation is leading the charge to ensure safe and effective therapies for all forms of CMT make it to the market to help those living with CMT today. We are working every day to support the families, the foundations, the academics, the scientists, the biotechs and the pharma companies who share our goal and who are working to make it happen.
Related News & Research
Dr. Yulia Grishchuk Joins CMTRF’s Scientific Advisory Board
CMT Research Foundation is pleased to announce that Dr. Yulia Grishchuk, Ph.D., has joined the Foundation’s Scientific Advisory Board. The SAB is composed of distinguished scientific and clinical experts specializing in Charcot-Marie-Tooth disease and drug...
CMT Research Foundation Invests in XtRNA Bio to Develop Gene Therapy for CMT2D
CMT Research Foundation has invested in a research project with XtRNA Bio aimed to develop a new viral gene therapy for Charcot-Marie-Tooth disease type 2D. How GARS1 Mutations Drive CMT2D CMT2D is caused by mutations in the GARS1 gene, which encodes glycyl-tRNA...
A Father Who Defies ‘Impossible’: Joao Cardoso’s Journey with CMT
Joao Cardoso, Brittany, France, Living with CMT Joao Cardoso never imagined that he would be riding through the town of Brittany, France, in a wheelchair, while his eight-year-old son, Edgar, pedals his bike alongside him. Because of Joao’s Charcot-Marie-Tooth...
Siemens’ Ann Fairchild Joins CMT Research Foundation Board of Directors
CMT Research Foundation is pleased to announce that Ann Fairchild, executive vice president and general counsel of Siemens USA, has joined the Foundation’s Board of Directors. Fairchild is a seasoned legal executive with more than 25 years of experience at Siemens,...
Applied Therapeutics Presents Findings From Phase 2/3 Clinical Trial of Govorestat in CMT-SORD
Applied Therapeutics recently presented findings from their INSPIRE Phase 2/3 clinical trial of govorestat (AT-007) for the treatment of Sorbitol Dehydrogenase Deficiency, a subtype of Charcot-Marie-Tooth disease, at the Peripheral Nerve Society’s 2025 Annual Meeting...
CMT Research Foundation Invests in Asha Therapeutics to Test Novel Drug for CMT2A
CMT Research Foundation has invested in a research project with Asha Therapeutics to investigate the therapeutic potential of inhibiting SARM1 (Sterile alpha and TIR motif-containing protein 1) using their novel drug, ASHA-624, for the treatment of Charcot-Marie-Tooth...
Vanderbilt University Researchers Identify Promising Compounds Targeting PMP22 Protein Expression, Paving the Way for New Treatments for CMT1A, CMT1E and HNPP
Researchers at Vanderbilt University have made significant progress in the development of small molecules aimed at treating Charcot-Marie-Tooth disease types caused by mutations in the PMP22 gene, which include CMT1A, CMT1E and hereditary neuropathy with liability to...
Because of Luca: A Family’s Fight Against CMT
Tara, Luca, Liam and Lane, Louisiana, Living With CMT When Luca Haik was just four years old, his mother, Tara, sensed something wasn’t right. “Luca came home from school every day crying in pain, rushing to take off his socks and shoes saying that his feet ‘felt...
Dr. Alessandra Bolino Unravels Complexities and Tests Treatment Strategies in CMT4B1
CMT4B1 is a severe form of Charcot-Marie-Tooth disease caused by mutations in the gene that makes Myotubularin-related protein 2 (MTMR2). Dr. Alessandra Bolino, PhD, discovered that mutations in this gene affect the production of fats, called phospholipids. Among...
OHio, What a Night: Lighting a Path to a Cure for Charcot-Marie-Tooth Disease
On Thursday, April 10, more than 120 patients, scientists, clinicians and pharma companies gathered in Columbus, Ohio for OHio, What a Night — an unforgettable evening dedicated to accelerating research and developing treatments and cures for Charcot-Marie-Tooth...
