CATEGORIES: Awareness | CMT Update | Inspire Community

Our CMT Inspire Community, Soon to be 2,000 Strong!

by | Jun 4, 2015 | 1 comment

HNF has partnered with Inspire to provide a safe online health and wellness community in which patients, families, friends, and caregivers connect with one another for support and information. Some of the HOT topics currently being discussed include “Pregnancy and CMT,” “Types of AFOs/Leg Braces,” and, “What You Wish Your Doctor Knew.”

If you have not joined us yet, please take a minute to set up your profile and join our community. Don’t forget to add your picture to make things a bit more personal. For more info on the CMT Inspire Community, please contact [email protected]

10 Reasons

Learn more on this topic

Related Blog Posts

Taking Matters Into Your Own Hands

by | Mar 9, 2015 | | 0 Comments

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:

Now GRIN is Accessible in 141 Different Languages!

Now GRIN is Accessible in 141 Different Languages!

by | Mar 9, 2015 | | 4 Comments

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.

Accelerating Patient Access to Investigational Drugs in 2015

Accelerating Patient Access to Investigational Drugs in 2015

by | Mar 9, 2015 | | 0 Comments

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.

Do You Have Charcot-Marie-Tooth Type 4 (CMT4)? – We Need Your Help

by | Mar 9, 2015 | , | 59 Comments

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.

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.

Scientific Advisory Board Meeting

Scientific Advisory Board Meeting

by | Mar 9, 2015 | , | 0 Comments

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.

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

by | Mar 6, 2015 | , , | 9 Comments

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.

Join the conversation

Leave a Comment

1 Comment

  1. chiefarchitect
    chiefarchitect on June 7, 2015 at 12:36 pm

    I am a 74 year old asian male that has had CMT approximately 8 years now. I was wondering if any other Asians have had similar experiences in the progression of the muscular weakness as well as blurred vision, hearing loss and speech problems. I think this website is a wonderful resource for those who suffer from the desease. Thank you.

    Reply

Submit a Comment

Your email address will not be published.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

Newsletter

Join for notifications on events, campaigns, & news