Paving pathways for mission critical projects

CMT Research Foundation is partnering with Dr. Maurizio D’Antonio of I.R.C.C.S. Ospedale San Raffaele, in Milan, Italy to fund the lab’s research of Myelin Protein Zero (MPZ), the gene associated with CMT1B.  Dr. D’Antonio’s lab is dedicated to studying the molecular mechanisms underlying the CMT group of diseases, naturally aligning with CMT Research Foundation’s mission in developing and delivering effective therapies for CMT.

CMT 1B is a demyelinating neuropathy, caused by mutations of the Myelin Protein Zero (MPZ).  Myelin provides electrical insulation to the nervous system and is frequently dysfunctional in CMT type 1.  With over 200 different mutations of MPZ, there are many reasons why the myelin dysfunctions.  These differences can cause people with CMT1B to have varied ages of onset, varied symptoms and varied mechanisms of action (what actually causes nerve degeneration).

Dr. Maurizio D’Antonio at Ospedale San Raffaele in Milan, Italy is widely revered as an expert in the biology of myelin and has a deep understanding of mutations of MPZ.   Dr. D’Antonio made several important discoveries in MPZ, including the identification of a disease mechanism which can be modulated thanks to a compound named IFB-088, currently in phase 1 clinical trial and that shall advance in phase 2 clinical trial for CMT1 through Inflectis BioScience.  

The CMT Research Foundation is funding a new project with Dr. D’Antonio which will test the genetic modulation of the same pathway in adult animal models with CMT1B, to determine if giving the compound later in life may modify the course of the disease.  If this genetic proof of principle is effective in animal models, the compound may be applicable to people who’ve suffered with symptoms of CMT1B for decades. Knowledge gained through funding of this project from the CMT Research Foundation could accelerate the advancement of clinical trials and possible approval of the treatment for adults with some mutations of
MPZ, also known as CMT1B.

CMT Research Foundation extends funding to deliver on our mission to accelerate the development and delivery of treatments and cures for CMT.   With gratitude and unwavering devotion, we are excited to embark on another mission critical project.  To learn more on how you can continue to support and further the development of CMT treatments, please visit our site for all research updates and news.  You may also donate now to fund this and many more treatment development efforts.