Pharnext and the Hereditary Neuropathy Foundation Collaborate to Increase Awareness of Charcot-Marie-Tooth Disease

Pharnext will provide financial support to several HNF’s initiatives including:

  • Enhanced design and development of a specific section in the HNF website featuring HNF newsletters, materials and latest news on CMT research (
  • Implementation of a video campaign during the “CMT Awareness” month in September 2014 in the US
  • Improvement of HNF’s CMT Support Community hosted on Inspire (
  • Expansion of the Global Registry for Inherited Neuropathies (GRIN) co-founded by HNF and Hannah’s Hope Fund (HHF) for Giant Axonal Neuropathy (GAN) (

GRIN has been developed by HNF and HFF to collect clinical and genetic information on patients diagnosed with the various forms of hereditary neuropathies such as CMT and GAN in order to advance therapies development for these debilitating disorders. Expanding GRIN further will allow researchers working on new therapies for hereditary neuropathies to access an invaluable source of data to move forward their research.

“HNF’s collaboration with Pharnext is an opportunity to further raise CMT awareness and to provide educational tools for patients, their families and researchers,” said Allison Moore, Founder and Chief Executive Officer, HNF.

“GRIN is a major initiative that will enable organizations such as Pharnext to more easily bring sorely needed treatments to those with hereditary neuropathies such as CMT thereby furthering HNF’s mission of raising awareness,” said Sean Ekins, CSO, HNF.

“Pharnext is supporting these efforts as we are dedicated to improving the lives of patients suffering from CMT. We look forward to the development of GRIN and the applications it will have to scientific studies such as our planned phase III trial of our investigational treatment for CMT 1A,” said Daniel Cohen, M.D., Ph.D., Chairman and Chief Executive Officer of Pharnext.

About CMT

Charcot-Marie-Tooth (CMT) disease, also known as Hereditary Motor and Sensory Neuropathy (HMSN) or Peroneal Muscular Atrophy (PMA), encompasses a heterogeneous group of inherited, progressive, chronic sensory and motor peripheral neuropathies. This group of disorders is characterized predominantly by distal muscle weakness and atrophy often associated with mild to moderate sensory loss, depressed tendon reflexes and high-arched feet. CMT is the most common inherited neuromuscular disorder. Overall prevalence of CMT is usually reported as 1 in 2500. Three different types of CMT disease are distinguished based on the abnormality that disrupts the nerve function: the demyelinating type (CMT1), the axonal type (CMT2), and an intermediate type. Further sub-classification is based on the underlying genetic mutation (e.g. PMP22 gene duplication for the CMT 1A subtype).

To date, no curative or symptomatic medications have been approved and treatment consists of supportive care such as orthotics, leg braces, physical and occupational therapy or surgery.

About Hereditary Neuropathy Foundation

Hereditary Neuropathy Foundation (HNF) is a non-profit 501(c)3 organization whose mission is to increase awareness and accurate diagnosis of Charcot-Marie-Tooth disease (CMT) and related inherited neuropathies, support patients and families with critical information to improve quality of life, and support research that will lead to treatments and cures. HNF and HHF last year launched the Global Registry for Inherited Neuropathies (GRIN) to collect clinical and genetic information on patients diagnosed with the various forms of inherited neuropathies in order to advance therapy development for these debilitating disorders.

To join the patient registry, visit

For further information, visit

About Pharnext

Pharnext is an advanced clinical stage biopharmaceutical company discovering and developing new therapeutics that target multiple key disease pathways for orphan and common neurological diseases including Charcot-Marie-Tooth disease, Alzheimer’s disease, amyotrophic lateral sclerosis and Parkinson’s disease. These new therapeutics consist of low dose combinations of repositioned drugs selected using Pharnext’s proprietary network pharmacology approach. The company’s first two lead programs are PXT-3003 for the treatment of Charcot Marie Tooth type 1A (phase 2 completed) and PXT-864 for Alzheimer’s disease (phase 2 ongoing) and other neurology indications. For further information, visit

Pharnext Contact

Catherine Scart-Gres, M.D.

Chief Medical Officer

HNF Contact

Allison Moore – Founder and Chief Executive Officer

Sean Ekins – Chief Scientific Officer

Hereditary Neuropathy Foundation

432 Park Avenue South, 4th Floor

New York, NY 10016

T: 212-722-8396, toll free 855-HELPCMT (435-7268)

F: 917-591-2758

Media Contacts 

Tony Russo, Ph.D.

Matt Middleman, M.D.

Russo Partners

T: 212-845-4251

T: 212-845-4272