CATEGORIES: Research
letter thank you

A Letter From HNF’s CEO/Founder

by | Dec 10, 2018 | 0 comments

letter

2018 was a pivotal year for the Hereditary Neuropathy Foundation (HNF) and the entire Charcot-MarieTooth (CMT) Community. Our team at HNF has worked tirelessly to ensure that we help to bring the first drug to market ASAP. As we wrap up 2018, we are challenging all of our generous HNF members, new and old friends, and others in the community to please consider supporting our work. We are 100% dedicated to developing and supporting treatments in 2019 and beyond. In fact, we are on the cutting-edge of more human trials within the next 12 months and our pre-clinical research is also looking promising for many types of CMT.

Our proudest accomplishment and most important initiative to accelerating therapies and early access to treatments was the successful execution of the Externally-led Patient-Focused Drug Development (PFDD) Meeting for the FDA for all types of CMT!

We could not have been successful without our donors’ support. HNF is transparent, collaborative and results driven. We will continue to work for you, bridge the gaps and partner only with leaders that will deliver more treatments in the near future.

Let’s continue the momentum toward our research goals to deliver more results for

hundreds and thousands of families living with CMT.

I ask you, from the bottom of my heart, to please make a donation (no amount is too small) as we wrap up 2018.

 

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Related Blog Posts

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.

New Study on CMT Type 2B

If we are to learn more about CMT and the effectiveness of rehabilitation it is worth asking the patient and their caregiver. A recent Italian study by Padua et al recently described a survey of CMT patients and caregivers and their perspectives and perceptions of rehabilitation efficacy and needs.

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”

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