Mary Chapman, features writer —

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

France-based Pharnext will use a capital raise of about 7.7 million euros ($8.4 million) to support the clinical development of PXT3003, the company’s experimental treatment for Charcot-Marie-Tooth disease type 1A (CMT1A). Led by existing investors, the funding included some U.S. institutional investors and Pharnext management members. “We…

To promote the value of exercise in Charcot-Marie-Tooth disease, the Hereditary Neuropathy Foundation is bringing its latest summit to central Florida. The free Movement is Medicine Summit Orlando will be held March 21 in Winter Park at the 80,000-square-foot, state-of-the-art Center for Health and Wellbeing, a…

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

The Institute for Gene Therapies (IGT) has launched, with the aim of maximizing the potential of gene therapies in genetic disorders such as Charcot-Marie-Tooth (CMT) disease. Comprised of industry leaders, scientists, and patient advocates, the IGT’s overarching goal is to set the foundation for a modernized regulatory…

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

With a $100,000 match pledge to CMT1A research, longtime Charcot-Marie-Tooth Association (CMTA) supporters Seth and Missy Warfield have issued a challenge to other members of the disease community. The pledge was in celebration of the nonprofit organization earning a prestigious four-star Charity Navigator rating. It’s the highest…

A special advisor to the Charcot-Marie-Tooth Association’s (CMTA) board of directors says he will match donations dollar-for-dollar up to $500,000 to support the organization’s research strategy to fight the disease he has — CMT2. Bruce Chizen was diagnosed in 2016, after decades of experiencing symptoms.

During Charcot–Marie–Tooth Awareness Month in October, the nonprofit CMT UK is aiming to increase knowledge about what it calls a common rare disease in the United Kingdom (UK). Charcot–Marie–Tooth disease (CMT) is thought to affect about 2.6 million people globally, including 25,000 in the UK, according…

In a research partnership with the CMT Research Foundation (CMTRF), a prospective treatment being developed by AcuraStem for amyotrophic lateral sclerosis (ALS) was found to be a promising preclinical therapy candidate for a form of Charcot-Marie-Tooth (CMT) disease. In the collaborative drug screen for…