hnf on the road

HNF On The Road

by | Dec 14, 2018 | 0 comments

HNF On the Road


Rare Disease Week on Capitol Hill

February 2018, Washington, DC  

  • HNF team and 10 CMT patients/caregivers participated in meetings with 12 Congress officials to inform them about Charcot-Marie-Tooth

Learn more here:


American Academy of Neurology Annual Meeting

April 2018, Los Angeles, CA  

  • Conducted important meetings with leading CMT stakeholders to enhance HNF’s programs  
  • Identified additional clinical sites to participate in the Pharnext PXT3003 Pediatric Trial to launch early 2019


Rare Patient Advocacy Symposium in Partnership with Penn Medicine Orphan Disease Center and Global Genes

May 2018, Philadelphia, PA

  • Allison Moore, a Rare Disease thought leader presented on “Patients as Key Opinion Leaders and Partners In Research and Clinical Development.” The presentation highlighted the vital role that patients play in the development of drugs and treatments for the CMT community


BIO International Convention

June 2018, Boston, MA  

  • Hosted by the Biotechnology Innovation Organization (BIO). BIO represents more than 1,100 biotechnology companies, academic institutions, state biotechnology centers and related organizations globally  
  • For the fourth year, HNF was invited to participate in the Patient Advocacy Pavilion to explore new TRIAD partnerships


Peripheral Nerve Society (PNS) Annual Meeting

July 2018, Baltimore, MD  

  • HNF was an exhibitor and connected with attending CMT physicians, researchers and partners from all over the world  
  • James Nussbaum, PT, PhD, SCS, EMT, oral poster, “Functional Improvements in Patients with CMT participating in Skilled Physical Therapy: A Retrospective Analysis”, in collaboration with HNF and the AlterG, was selected for display


HNF was honored to be chosen to present at two sessions at Global Genes Annual RARE Patient Advocacy Summit

October 2018, Irvine, California.  

  • Session – Architecting Your Disease Community HNF Advocacy Director Joy Aldrich moderated the session with Allison Moore, CEO of HNF and Jill Jarecki, PhD and CSO of Cure SMA, on challenges and benefits of engaging in the effort to help FDA and other stakeholders in understanding the patients’ disease experience to accelerate therapy development.  
  • Closing Session – Data and Technological Innovation Allison Moore, CEO of HNF presented along with Scott Schliebner, VP Scientific Affairs, PRA Health Sciences, a leading CRO company, to close the meeting on the importance of utilizing innovative technology (Voice Activated Technology and conducting virtual clinical trials) and how these technologies will improve drug discovery, clinical trials design and the acceleration of patient screening and recruitment.


Peer Support Boot Camp hosted by Child Neurology Foundation (CNF)

October 2018, Irvine, CA.  

  • HNF continues our collaboration with the CNF and the Family Support & Empowerment Program (FSEP) to bring peer support to the CMT caregiver community. Stay-tuned!


HNF Hosted CMT Symposium at the annual American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Conference

October 2018, National Harbor, MD

  • A 90-minute educational session moderated by Estela Lugo, HNF Medical Outreach Director featured a panel of experts with insight on the importance of Patient Reported Outcomes in new technology, genetic testing, therapy development, patient care, and Healthcare Provider networks.
  • Panelists included: Mark Larkin, PhD, Vitaccess Founder, Andrea Paal, MS, CGC, Athena Diagnostics, Florian Thomas, MD, MA, PhD, MS, Chair of the Neuroscience Institute and the Department of Neurology, and Director of the Hereditary Neuropathy Foundation Center of Excellence at Hackensack University Medical Center, Jahannaz Dastgir, DO – Pediatric Neurologist at Atlantic Health, James Nussbaum, PT, PhD, SCS, EMT, founder and director of ProHealth & Fitness.


Watch our AANEM full presentation click here:

We are proud to announce that HNF’s AlterG Anti-gravity Clinical Study Principal Investigator Dr. Nussbaum received a President’s Research Initiative Award at the AANEM.

Learn more on this topic

Related Blog Posts

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.

Support CMT Therapeutic Alliance

HNF has entered into a joint venture – the CMT Therapeutic Alliance – with a unique non profit organization (BioPontis Alliance for Rare Diseases) that brings professional drug discovery capabilities to translate our research results into potential treatments.

Sneak Peak of Fall Newsletter: CMT Research Study Survey

My name is Elizabeth Francisco and I am a graduate student from the Genetic Counseling program at the University of North Carolina Greensboro. I am inviting you to participate in a research study. The goal of my study is to learn more about the experiences of people with Charcot-Marie-Tooth (CMT) with genetic counseling and genetic testing. Adults with CMT and parents or legal guardians of someone of any age who has a diagnosis of CMT are eligible to participate

Building Awareness on CMT and Supporting the Patient Community – HNF Announces Support from Pharnext

HNFpharnext recently entered into a partnership with the French biopharmaceutical company, Pharnext, to help raise awareness of Charcot-Marie-Tooth (CMT) disease and support the CMT patient community through several initiatives. Building awareness is key! Pharnext’s support will assist HNF in distributing HNF’s CMT Update quarterly newsletter, enhancing the Global Registry for Inherited Neuropathies (GRIN), setting up activities for CMT September Awareness Month in the US and strengthening the CMT Inspire Community.

Quality of Life and CMT Research

Did you know that 95% of clinical trials fail? There are multiple causes, most related to efficacy or safety, which obviously can be harmful and risky for patients. The risk-reward of enrolling in trials is a judgment call based on the devastating effects of disease related to quality of life (QoL) or life-threatening disease. With CMT, the risk-reward is more of a challenging question for many, as CMT in most cases is non-fatal.

Help us answer questions that your doctors and the CMT Research Community aren’t too sure about.

Did you know that you can become part of a community in therapy development and further research for all forms of CMT and inherited neuropathies? The mission of the Global Registry for Inherited neuropathies (GRIN) is to collect clinical and genetic information from patients with ALL forms of Charcot-Marie-Tooth (CMT) and other related rare and ultra rare inherited neuropathies.

Hot off the press

It seems like almost weekly there is another new publication on CMT with interesting basic biology. While an earlier “Hot off the press” highlighted the work of Cherry and co-workers at the University of Texas Southwestern Medical Center who showed show that neurons lacking a gene for rab7 result in neuropathy.

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