CATEGORIES: Research
MDBR Pilot Grant Program

Orphan Disease Center Million Dollar Bike Ride Pilot Grant Program

by | Aug 23, 2019 | 0 comments

Calling all Charcot-Marie-Tooth Researchers!

team cmt bikeThe ODC MDBR Pilot Grant Program provides a one‐year grant to support research related to a rare disease represented in the 2019 Million Dollar Bike Ride. HNF reached its $30,165 goal which was generously matched by the Orphan Disease Center. We want to thank all our riders and donors for participating. The grant will be a 1 year award with an optional 6 month no cost extension for a one time award of $60,330 starting January 1, 2020.

 

 

Eligibility

All individuals holding a faculty‐level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA.

Letter of Interest Instructions:

Please visit the ODC website (link below) to submit your Letter of Interest (LOI). This one-page LOI is due no later than Monday, September 23, 2019 by 8pm (EST).

Click RFA guidelines and scroll down to #7 for details for CMT grant.

Full Application Instructions and Review Procedure

NOTE: Full Application is by invitation only after review of Pre-Application 

Proposal Due Date: Thursday, October 24, 2019 no later than 8pm (EST). Full application documents are to be uploaded on our website, by invitation only.

Apply Today

Learn more on this topic

Related Blog Posts

A New Mouse Model for Charcot-Marie-Tooth (CMT2)

by | May 27, 2015 | , , | 0 Comments

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

by | Mar 9, 2015 | , , | 53 Comments

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

by | Mar 9, 2015 | , | 0 Comments

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

by | Sep 16, 2014 | , | 2 Comments

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

by | Aug 19, 2014 | , , | 1 Comment

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

by | Jul 7, 2014 | | 4 Comments

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.

by | May 19, 2014 | | 0 Comments

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

by | Feb 28, 2014 | | 0 Comments

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.

Join the conversation

Leave a Comment

0 Comments

Submit a Comment

Your email address will not be published.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

Newsletter

Join for notifications on events, campaigns, & news