News & Stories
See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.
CMTRF Welcomes Dr. Grace Pavlath as Chief Science Officer
CMT Research Foundation (CMTRF), a not-for-profit organization focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, has added Grace Pavlath, Ph.D. to the team as the new Chief Scientific Officer. CMT is a progressive genetic nerve disease...
New research efforts by Neurogene, give hope to patients suffering with an ultra-rare type of CMT: CMT4J
Neurogene, a leading company developing genetic medicines for people with rare, devastating neurological diseases, announced today that it is recruiting patients for a natural history study for CMT4J. "Key data now being collected to support endpoint assessment for...
Is “Disease-In-A-Dish” in Combination with Animal Models A Faster Track to a Cure for CMT?
From time to time the leadership of CMTRF will explore the challenges of finding treatments and a cure for CMT. To the extent we are able, we will try to overcome these obstacles to accelerate successful CMT research. Treatment of diseases, especially rare diseases...
Scenes from BIO2019
Last week Paul August and I attended the BIO International Convention, a conference with over 16,000 leaders from the biotech and pharmaceutical industry. We met a number of companies interested in advancing drug development for CMT. There are four primary factors...
Partnerships and Priorities: The Pathway to CMT Results
From time to time the CMT Research Foundation will examine the impediments to finding a cure for CMT. As the Chairman of a nonprofit foundation whose ONLY mission is to fund research that will lead to a cure for Charcot-Marie-Tooth disease (CMT), I am often...
New Evidence for Gene Replacement in a Mouse Model of Charcot-Marie-Tooth Disease
In an article published yesterday in Neurology Today, Dr. Kleopa demonstrated that gene replacement could be a viable treatment for recessive forms of CMT. CMT type 4C is a demyelinating disease, and this treatment both decreased the number of unmyelinated fibers and...
CMTRF Responds to FDA Fast Track Designation of PXT3003
The FDA Grants Fast Track Designation to Pharnext's PXT3003 for CMT1A Pharnext, an advanced clinical-stage biopharmaceutical company, created a drug combination of three drugs: baclofen, naltrexone and sorbitol known as PXT3003. Baclofen is known to relieve muscle...
CMTRF investigates a gene therapy program for CMT1A
CMT Research Foundation Partners with Cyprus Institute for Neurology and Genetics’ Kleopas Kleopa to Launch Study of Possible Gene Therapy for CMT1A This is the second major investment by CMTRF. ATLANTA (January 16, 2019) The newly launched CMT Research Foundation...
The Inherited Neuropathy Consortium (INC) welcomes the CMT Research Foundation as a partner in their Patient Advocacy Groups
Today the Inherited Neuropathy Consortium (INC) named the CMT Research Foundation as a partner in their Patient Advocacy Groups. This gives the CMT Research Foundation access to INC resources, researchers and its clinical infrastructure through 21 CMT Centers of...
Is there a connection between CMT1X and Multiple Sclerosis?
A recent study by Georgios Koutis et al at the National and Kapodistrian University of Athens recently published a paper in The Journal of Neurology, Neurosurgery and Psychiatry suggesting emerging evidence of a connection between CMTX1 and Multiple Sclerosis (MS)....
Important Research News Announcements
- Genomic Studies in Charcot-Marie-Tooth Disease July 31, 2024
- Charcot-Marie-tooth disease type 2A: An update on pathogenesis and therapeutic perspectives March 5, 2024
- Accelerate Clinical Trials in CMT (ACTCMT) Study March 1, 2024
- Creation of a Schwann Cell Gene Regulatory Network February 29, 2024
- Activation of XBP1s attenuates disease severity in models of proteotoxic Charcot-Marie-Tooth type 1B [Preprint] February 2, 2024
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