CATEGORIES: CMT Summit | Research
rare patient advocacy symposium

Allison Moore Speaker at 2018 Rare Patient Advocacy Symposium

by | Jul 26, 2018 | 0 comments

allison mooreOn May 19, the Hereditary Neuropathy Foundation (HNF) participated in the Rare Patient Advocacy Symposium in partnership with Penn Medicine Orphan Disease Center and Global Genes. HNF’s CEO/Founder, Allison Moore, spoke on the importance of the “Patient’s Voice” and “Patients as Key Opinion Leaders and Partners In Research and Clinical Development”.

The presentation highlighted the vital role the patients play in the development of drugs and treatments for the CMT community. Moore presented the key steps and processes used to acquire our upcoming Externally-led Patient-Focused Drug Development (PFDD) meeting with FDA officials and other stakeholders, as well as the tools HNF will use to inform the FDA on what matters most to patients and how they view the benefits and risks of potential treatments.

Attendees were also briefed on the wide spectrum of Charcot- Marie-Tooth (CMT) symptoms and severities, as well as on the urgency for collecting patient data and the importance of natural history studies. HNF’s innovative proprietary patient registry — Global Registry for Inherited Neuropathies (GRIN), will help to improve drug discovery, clinical trials design and the acceleration of therapy development and regulatory approvals. HNF has been collecting patient data for close to 5 years, part of the organization’s continued commitment to making patients a key part of the research process and clinical development.

Moore has extensive experience in patient engagement methods, with an emphasis on identifying the gaps that have been hindering CMT patient care, standard of care guidelines, issues with patients having delayed and accurate diagnosis, and the need for improved clinical outcome measures and endpoints to support clinical trials.

The symposium was another valuable opportunity to network and partner with like-minded advocacy groups and key players to exchange valuable resources. HNF is dedicated to remaining on the cutting-edge of new pathways, technologies and collaborations like those displayed at the symposium. HNF is grateful to Global Genes for inviting us to participate!

We will continue to work tirelessly to provide the optimal treatment path for our CMT families, and look forward to meeting many of you at our “Voice of the Patient” Weekend, Sept 28-29.

Learn more on this topic

Related Blog Posts

Allison Moore is going to be published!

HNF’s CSO (Chief Scientific Officer), Sean Ekins wrote a blog about his work with Allison Moore and her two “fighter Mom” friends Lori Sames and Jill Wood. He named his blog: “Rare disease heroes – Extraordinary collaborators we should be listening too.” Sean helped my friends and I write a paper called: “Multifaceted roles of ultra rare and rare disease patients/parents in drug discovery.” YES, Allison Moore is going to be published! It will be in Drug Discovery Today, soon. The link to the reprint is below.

HNF-WVU-NIOSH Study update:

We are pleased to update our readers on the progress of the WVU-NIOSH study, “A Bi-Directional Translational Model of Exercise Training in the Treatment of Charcot-Marie-Tooth Disease”

Dr. Robert Baloh Answers Our Questions About iPS Cell Research

Big news is happening on a cellular level at Cedars-Sinai Medical Center’s Regenerative Medicine Institute in Los Angeles, CA.  Robert H. Baloh, MD PhD, Director of Neuromuscular Medicine, in conjunction with Patrick D. Lyden, MD Neurology Dept. Chair, and Clive Svendsen, PhD, Institute Director, are studying disorders that start in nerve cells. 

Join the conversation

Leave a Comment


Submit a Comment

Your email address will not be published. Required fields are marked *


Join for notifications on events, campaigns, & news