Throughout 2018, HNF represented the CMT community at major scientific, medical, and advocacy conferences around the world. Here is a summary of where we went and what we accomplished.

Rare Disease Week on Capitol Hill

February 2018, Washington, DC

The HNF team and 10 CMT patients and caregivers met with 12 members of Congress to educate them about Charcot-Marie-Tooth disease and the needs of the CMT community. Learn more about Rare Disease Week.

American Academy of Neurology Annual Meeting

April 2018, Los Angeles, CA

HNF held important meetings with leading CMT stakeholders to strengthen HNF’s programs and identified additional clinical sites to participate in the Pharnext PXT3003 Pediatric Trial, which was expected to launch in early 2019.

Rare Patient Advocacy Symposium

May 2018, Philadelphia, PA

Presented in partnership with Penn Medicine’s Orphan Disease Center and Global Genes, this symposium featured Allison Moore, a rare disease thought leader. Her presentation, “Patients as Key Opinion Leaders and Partners in Research and Clinical Development,” highlighted the vital role that patients play in drug and treatment development for the CMT community.

BIO International Convention

June 2018, Boston, MA

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

Peripheral Nerve Society (PNS) Annual Meeting

July 2018, Baltimore, MD

HNF exhibited at the PNS Annual Meeting and connected with CMT physicians, researchers, and partners from around the world. James Nussbaum, PT, PhD, SCS, EMT, presented an oral poster in collaboration with HNF and AlterG titled “Functional Improvements in Patients with CMT Participating in Skilled Physical Therapy: A Retrospective Analysis.”

Global Genes Annual RARE Patient Advocacy Summit

October 2018, Irvine, CA

HNF was honored to present at two sessions:

  • Architecting Your Disease Community: HNF Advocacy Director Joy Aldrich moderated a session with Allison Moore, CEO of HNF, and Jill Jarecki, PhD, CSO of Cure SMA. The panel discussed the challenges and benefits of engaging with the FDA and other stakeholders to advance understanding of the patient disease experience and accelerate therapy development.
  • Closing Session: Data and Technological Innovation: Allison Moore presented alongside Scott Schliebner, VP of Scientific Affairs at PRA Health Sciences, on the importance of innovative technology, including Voice Activated Technology and virtual clinical trials, for improving drug discovery, clinical trial design, and patient screening and recruitment.

Peer Support Boot Camp (Child Neurology Foundation)

October 2018, Irvine, CA

HNF continued its collaboration with the Child Neurology Foundation (CNF) and the Family Support and Empowerment Program (FSEP) to bring peer support resources to CMT caregivers.

HNF CMT Symposium at AANEM

October 2018, National Harbor, MD

HNF hosted a 90-minute educational session at the annual American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) conference, moderated by Estela Lugo, HNF Medical Outreach Director. The panel focused on Patient Reported Outcomes and their applications in genetic testing, technology, 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 HNF Center of Excellence at Hackensack University Medical Center; Jahannaz Dastgir, DO, Pediatric Neurologist at Atlantic Health; and James Nussbaum, PT, PhD, SCS, EMT, founder and director of ProHealth and Fitness.

HNF is also proud to report that Dr. Nussbaum, Principal Investigator of HNF’s AlterG Anti-Gravity Clinical Study, received a President’s Research Initiative Award at AANEM.

Watch the full AANEM presentation.