Mary Chapman, features writer —

The first patient has been enrolled in what’s regarded as the first natural history study of Charcot-Marie-Tooth disease type 4 (CMT4J), with the ultimate goal of one day developing a gene therapy for the disease, announced the study’s collaborators, Neurogene and UT Southwestern Medical Center. The study’s…

Some $10 million in National Institute of Neurological Disorders and Stroke (NINDS) grants aimed at accelerating clinical trials in rare neurological disorders will support a Charcot-Marie-Tooth (CMT) disease biomarkers project. Funding comes from NINDS’ Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases program, established to back investigations…

Because Charcot-Marie-Tooth (CMT) disease ran in her family, Denise Snow suspected in high school that frequent falls were more than clumsiness. “I recognized the disease in myself,” said Snow, 63, after a Charcot-Marie-Tooth Association (CMTA) patient conference in Detroit. “As time went on, symptoms increased. I started…

The Charcot-Marie-Tooth Association (CMTA) has granted $1 million to scientists searching for treatments, but it could cost up to $500 million to bring a viable disease therapy to market, according to the organization’s CEO, Amy Gray. Speaking April 27 to more than 100 people at CMTA’s Patient & Family…

The Charcot-Marie-Tooth Association (CMTA) has awarded approximately $1 million in grants to scientists worldwide investigating treatments and a cure for the disease of the peripheral muscle-controlling nerves. The grants bring to more than 50 the number of Strategy to Accelerate Research (STAR) projects the association is funding, according…

Several Charcot-Marie-Tooth Association (CMTA) events are planned in the coming months for Charcot-Marie-Tooth (CMT) patients and caregivers. The organization is hosting a live Facebook webinar March 26 at 9 p.m. EDT. It will feature Kate Lair, a member of the CMTA advisory board and former claims adjudicator. The…

To better understand the effect of Charcot-Marie-Tooth (CMT) disease on daily living, an Elizabethtown College student is calling for young adults with the disorder to participate in a research study. Allison Eveler, a senior occupational therapy major, is conducting her undergraduate research on CMT’s impact on the social…

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…

The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…

 To help it reach its fundraising goal by year’s end, the Charcot-Marie-Tooth Association (CMTA) has issued its 35th Anniversary $150,000 Research Challenge. Donations will be matched dollar for dollar through Dec. 31, 2018. Fundraising totals are continually updated on the CMTA website.  Funds will go toward investigations…