HNF is excited to hold the 2nd Annual Patient-Centered Summit with a focus this year on CMT/HNPP and Pain in Cambridge, MA! The Summit will be held on the campus of the Massachusetts Institute of Technology (MIT) in the Samberg Conference Center.
Registration opening soon. Click here for information more information.
Joy joined HNF in February 2015 to focus on the growth of HNF’s online patient support community as a CMT Advocacy Director. Joy and her husband, Toby, live in Seattle, WA, with their beloved yellow lab, Charley.
Corporate matching gift programs are charitable giving programs setup by corporations in which the company matches donations made by employees to eligible nonprofit organizations. For example, if a donor works for Bank of America and donates $100 to the Hereditary Neuropathy Foundation, Bank of America will double the donation by also writing a check for $100.
Do you have a rare form of CMT?
We were contacted recently from a patient with CMT2D who was looking for advice on how to make a difference. Our recommendations for all people with an ultra-rare form of CMT who want to get involved:
The Global Registry for Inherited Neuropathies (GRIN) has implemented Google Translate. Why did we choose Google? Because Google is leading the way in teaching computers how to interpret meaning, avoiding the traditional method of decoding language.
Currently the FDA is working to update the process for physicians applying for accelerated patient access to investigational drugs, while the drug or biological product is being tested in clinical trials. This will also be important for the CMT community as clinical trials for this disease are being launched.
Autosomal recessive demyelinating CMT4 patients present with earlyonset and slowly progressing symptoms. These include progressive distally accentuated weakness and atrophy of muscles in the lower limbs. In addition, patients may have weakness and atrophy of hands, sensory loss and pes cavus (high-arched feet), and walking difficulties. Further information on CMT4 can be found at Orphanet.
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.
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.
On the 7th of November we convened our scientific advisory board meeting at the HNF offices in NY. We have written a detailed review that has been published and captures all of the discussion and make this freely available to the scientific community.
We are creating an innovative HCP Directory which provides these clinicians with additional tools to care for their patients.
Does anyone know the difference between CMT and HNPP. I have read that they come under the same umberella. I have HNPP and so do two of my sisters. We have a wide range of symptoms, with various disabilities. I need a walking stick to move about and most days I can’t walk more than a few metres. My elder sister, who was first diagnosed with HNPP, 20+ years ago, can walk around and goes on holiday two ot three times a year. I would be interested to find out about the difference and similarities in the two diseases.
Thank you.
Carol.