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.

Is “Disease-In-A-Dish” in Combination with Animal Models A Faster Track to a Cure for CMT?

Jul 1, 2019 | CMT Research Updates, Press Releases, Research News

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

Jun 11, 2019 | News

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

May 31, 2019 | CMT Research Updates, Press Releases, Research News

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

May 24, 2019 | CMT Research Updates, Press Releases, Research News

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

Feb 11, 2019 | CMT Research Updates, News

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

Jan 17, 2019 | CMT Research Updates, CMTRF Funded Research, Press Releases

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

Jan 9, 2019 | CMT Research Updates, Press Releases

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?

Dec 18, 2018 | About CMT, CMT Research Updates

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)....

Two papers were recently published regarding CMT1B and Axonal Neuregulin 1 Type III (Nrg1TIII), a protein which builds myelin (the insulation) on the nerves.

Dec 14, 2018 | CMT Research Updates

In summary, there is a molecule which blocks the Nrg1TIII in CMT1B, causing the demyelination of the nerves. Both papers found that if the strength or amount of Nrg1TIII can be controlled, the nerves affected by CMT may be restored. When more of the protein is added...

Why FDA acceptance of gene therapy in SMA matters to CMT

Dec 7, 2018 | CMT Research Updates

Novartis's announcement about FDA filing acceptance could translate to CMT On Monday, Novartis, the parent company of AveXis, announced that a license application for their gene therapy approach to treat Spinal Muscle Atrophy (SMA) was accepted by the FDA. This is a...