CATEGORIES: CMT Update

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

by | Aug 19, 2014 | 1 comment

GRIN

Dr. Sean Ekins is Principal Investigator of GRIN, which is now in its second year. Dr. Ekins is a clinical pharmacologist by training with over 18 years of experience in pre-clinical drug development, toxicity and computational drug discovery research in large Pharma (Lilly, Pfizer), biotech, informatics software companies (GeneGo, Collaborative Drug Discovery, Inc.) and start up drug companies (Phoenix Nest).

As Chief Science Officer of HNF, it is his responsibility to oversee the success of our TRIAD research program in which we fund translational research (pre-clinical and clinical),  projects. HNF’s goal is to expedite the research process from bench to bedside. While there has been a considerable amount of research on CMT there is urgency to provide the thousands of patients progressing rapidly, with a therapeutic that offers a benefit to them.

As we continue to translate our research and partner with various academics, industry leaders, government and not-for-profit stakeholders that are aligned with HNF’s mission, your participation is an intricate and an important part of the process. Please Join us and contribute to GRIN, our patient registry, which will help us in our quest to cure CMT.

Learn more on this topic

Related Blog Posts

Breaking News: First Therapeutic Gene Therapy to Treat an Inherited Neuropathy is Approved for Clinical Trial!

The first disease community to receive a therapeutic gene to the spinal cord for an ultra rare inherited neuropathy is Giant Axonal Neuropathy (GAN). Congratulations to Hannah’s Hope Fund (HHF), a 501(c)3 public charity, which has driven this collaborative research in less than six years. Six million dollars has been raised to date to fund pre-clinical and clinical research on this rare disease.

A Rare Disease Patient That Does It All!

A movie script could not play out like this. There are very few occasions when a rare disease patient becomes the topic of a story that is truly uplifting. Often the stories are tragic or disheartening. That was not the case, however, with CMT patient Kim Goodsell.

Hot Off the Press

One of the ways that some CMT patients first become aware of their disease is when they are given a drug treatment for another disease. This is termed chemotherapy-induced neurotoxicity. Drugs such as paclitaxel and the vinca alkaloids that are widely used in cancer treatment cause severe peripheral neuropathy and in some patients this exacerbates CMT, revealing it perhaps for the first time.

Kara Q&A: How to “support” a friend or family member that’s affected with Charcot-MarieTooth.

My name is Courtney Hollett, Fundraising Coordinator at the Hereditary Neuropathy Foundation. This time of year I count my blessing daily and wanted to share with you a Q&A session I had with a new supporter of HNF. Kara, like myself has many family members affected with CMT and I reached out to her to share her thoughts and advice about how to “support” a friend or family member that’s affected with Charcot-MarieTooth.

Collaboration is the Key to HNF Success

Collaboration is the Key to HNF Success

We at HNF are so proud of the accomplishments of those scientists we fund and are asking you to please continue to support our efforts. Our Therapeutic Research In Accelerated Discovery (TRIAD) program is a proven collaborative model in the drug discovery process.

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

Join the conversation

Leave a Comment

1 Comment

  1. J Brian C Saunders

    Could you please give me a definition of CMT type 2 axonal
    Thanking you in anticipation

    BRIAN

    Reply

Submit a Comment

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

Newsletter

Join for notifications on events, campaigns, & news