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.
Blog Category
Research
Total posts in Category: 91
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
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.
CMT Research Update: The Drug Discovery Process
Learn more about the process of drug discovery and bringing a drug to market.
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.
Using Zebrafish to Search for Therapeutics for CMT2A
Research on CMT is global, and covers both laboratory and clinical studies. It is critically important to be aware of what is happening elsewhere as well as in the USA because it can have implications for what we do and fund at the HNF.
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.
New Registry Helps Researchers Find A Cure: Q & A with CEO Kyle Brown, Crossroads
The Hereditary Neuropathy Foundation (HNF) and Hannah’s Hope Fund (HHF) are excited about their partnership to co-fund the Global Registry for Inherited Neuropathies
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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Research (91)
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Awareness (32)
Centers of Excellence (13)
CMT en Español (1)
CMT Summit (52)
CMT Update (74)
CMT-Connect (30)
CMT-Connect Webinars (10)
Webinars – Accessible Travel (3)
Webinars – Adaptive Driving (2)
Webinars – Bracing (2)
Webinars – Breathing (1)
Webinars – Canine Companions (1)
Webinars – Cannabis (3)
Webinars – Dating and Relationships (3)
Webinars – Family Planning (1)
Webinars – FEATURED (7)
Webinars – Genetic Testing (1)
Webinars – Home Accessibility (2)
Webinars – Inclusive Employment (3)
Webinars – Inspire Community (1)
Webinars – Legal Rights and Benefits (3)
Webinars – Newly Diagnosed (2)
Webinars – Nutrition (1)
Webinars – Pain (5)
Webinars – Podcasts (1)
Webinars – PT and Exercise (9)
Webinars – Research and Clinical Trials (9)
Webinars – School and College (4)
Webinars – Surgery (2)
Webinars – Telemedicine (1)
Webinars- Emotional and Mental Health (5)
Community (3)
Emerging Technologies (1)
Faces of CMT (1)
Faces of CMT – Addie (2)
Faces of CMT – Bernadette (9)
Faces of CMT – CMT4A (7)
Faces of CMT – Grace (18)
Faces of CMT – HELP Fund (8)
Faces of CMT – HNPP Fund (3)
Faces of CMT – James (1)
Faces of CMT – Jaxson (7)
Faces of CMT – Zach (18)
Featured (28)
GRIN Patient Registry (39)
HNF Team (10)
Industry (3)
Living with CMT (77)
Medical professionals (2)
Movement Is Medicine (17)
Optic Hereditary Neuropathy (5)
Our Impact (3)
Clinical Trials (17)
PFDD (1)
PFDD Meeting 28SEP2018 (5)
Past Events (35)
Patience Resources (0)
Accessible Travel (4)
Bracing (12)
Breathing (3)
Canine Companions (5)
Cannabis (8)
Caregivers (3)
Dating and Relationships (7)
Emotional and Mental Health (8)
Family Planning (1)
Fashion and Products (1)
Genetic Testing (13)
Home Accessibility (2)
Inclusive Employment (3)
Inspire Community (7)
Legal Rights and Benefits (8)
Neurotoxic Drugs (4)
Newly Diagnosed (8)
Nutrition (3)
Occupational Thearpy (2)
Pain (8)
Podiatry (4)
PT and Exercise (25)
Research and Critical Trials (27)
School & College (6)
Surgery (10)
Telemedicine (1)
Tips and Hacks (2)
Patient-Focused Research (1)
Pediatrics & CMT (9)
Registry (15)
Research (91)
School Outreach Program and Team CMT Kids (1)
Special Events (1)
Bike New York (19)
Other Events (4)
Spin-a-thon (7)
TCS New York City Marathon (7)
Team CMT Members (60)
The CMT Genie (7)
TRIAD (23)
TRIAD – Clinical Trial Readiness (3)
Vitaccess (1)
TRIAD – Research Gifts (0)
University of Helsinki (2)
Veneto (1)
TRIAD – Sponsored Research (0)
Burke Insitute (3)
University of Cambridge (1)
University of Miami (1)
University of San Antonio Texas (1)
TRIAD – Therapeutics (3)
Applied Therapeutics (9)
Pharnext (10)
Rarebase (4)
Types of CMT (1)
CMT Type – CMT1A (20)
CMT Type – CMT1B (1)
CMT Type – CMT1X (1)
CMT Type – CMT2A (7)
CMT Type – CMT2C (4)
CMT Type – CMT4A GDAP1 (3)
CMT Type – CNTNAP1 (1)
CMT Type – GAN (5)
CMT Type – HDAC6 (1)
CMT Type – HNPP (3)
CMT Type – MTRFR-C12orf65 (5)
CMT Type – SORD Deficiency (8)
Upcoming Events (5)
Ways to Give (4)
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