HNF to host an educational CMT symposium at AANEM’s Annual Conference October 10-13th in Washington, D.C.
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Research
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HNF Presents at RARE Patient Advocacy Summit Hosted by Global Genes®
The Annual RARE Patient Advocacy Summit, hosted by Global Genes, is in its seventh year and will take place on October 3-4 at the Hotel Irvine in Irvine, California.
Allison Moore Speaker at 2018 Rare Patient Advocacy Symposium
On May 19, HNF participated in the Rare Patient Advocacy Symposium in partnership with Penn Medicine Orphan Disease Center and Global Genes.
Neurology Reviews® & NORD Publish 2018 Rare Neurological Disease Special Report
The 2018 Rare Neurological Disease Special Report has been published by Neurology Reviews in collaboration with NORD.
$200,000 In Donations From True Reply Supports CMT Research
True Reply and HNF have collaborated to enhance patient focused research and knowledge of what matters most to patients living with CMT.
StarWise Therapeutics LLC & HNF Join Forces for Treatment of CMT2A
StarWise Therapeutics LLC & HNF join forces to expedite the advancement of a new drug entity to the clinic for the treatment of CMT2A.
Muscle Cramp Study Underway
In October 2017, Flex Pharma initiated a Phase 2 study of CMT, referred to as the COMMIT study.
Exciting Corporate News: Invitae, A Genetic Information Company, Acquires AltaVoice
Invitae, A Genetic Information Company, Acquires AltaVoice, HNF’s Global Registry for Inherited Neuropathies (GRIN) Platform Host
New Collaboration with the University of North Carolina at Chapel Hill and The Jackson Laboratory Addresses Charcot-MarieTooth/Inherited Neuropathy Mutations Using Gene Therapy
HNF’s initial gene therapy work will focus on CMT type 6, which is caused by a recessive mutation in the C12orf65 gene and is currently no treatment.
A Bi-Directional, Translational Model of Resistance-Type Exercise Training in the Management of Charcot-Marie-Tooth (CMT) Disease
A team of government researchers, including Dr. Robert Chetlin, have collaborated with Dr. Michael Sereda and Dr. Klaus Nave of the Max Planck Institute for Experimental Medicine (MPI) to successfully secure the CMT1A transgenic rat from MPI and establish a colony in the United States.
A New Mouse Model for Charcot-Marie-Tooth (CMT2)
We were recently informed that The Jackson Laboratory (JAX, a nonprofit biomedical research institution headquartered in Bar Harbor, Maine) had taken delivery and will be distributing a newly generated CMT-related mouse model. The new model expresses mutant mitofusin 2, a mitochondrial membrane protein involved in mitochondrial fusion and regulation of vascular smooth muscle cell proliferation.
Hot Off the Press – Potential Treatment for CMT1A
Two recent publications from Pharnext describe a novel synergistic combination of 3 drugs (baclofen, naltrexone and sorbitol) and its effect on CMT1A both in the lab and in a phase II clinical trial. These 3 drugs already approved but for unrelated conditions, are combined at new optimal lower doses and under a new formulation. This novel potential therapeutic is called PXT-3003.
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