April 14, 2021April 14, 2021
Gene Deficiency in CMTX May Limit Nerve Injury, Mouse Study Suggests
News
Sciatic motor nerves in mice lacking the GJB1 gene — defective in people with X-linked Charcot-Marie-Tooth (CMTX) disease — regenerated ... Read more
April 13, 2021April 9, 2021
Receiving an Offer of Help Reminded Me What It Means to Be a CMTer
Columns, On My Own Two by Young Lee
I had just exited the county building after getting my second shot of the COVID-19 vaccine and was making my ... Read more
April 9, 2021April 9, 2021
$20,000 Grant Will Help Unveil How Certain Mutations Fuel CMT
News
Scientist Timothy Hines, PhD, was awarded a grant of $20,000 to establish cell models to study Charcot-Marie-Tooth (CMT) disease at ... Read more
April 7, 2021April 7, 2021
Pharnext Enrolls 1st Patient in PREMIER Trial Testing PXT3003 for CMT1A
News
The first patient has been enrolled in a pivotal Phase 3 trial testing Pharnext’s investigational therapy PXT3003 for Charcot-Marie-Tooth disease type ... Read more
April 6, 2021April 2, 2021
Home Cooking Videos Showed a New Side of CMT Symptoms
Columns, On My Own Two by Young Lee
I’m no Gordon Ramsay. And it’d be insulting to actual professionals if I were to call myself a chef or ... Read more
April 2, 2021April 2, 2021
Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds
News
As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an ... Read more
March 31, 2021March 31, 2021
Interim Results of CMT&Me Study Presented in Webinar
News
Depression, hand-and-finger weakness, and a lengthy delay from symptoms to diagnosis contribute to the burdens felt by people living with ... Read more
March 26, 2021March 26, 2021
CMTA Grants Researchers $1.1M to Advance Trials in CMT1X, CMT2A
News
The Charcot-Marie-Tooth Association (CMTA) has awarded $1.1 million to help scientists advance clinical trials evaluating potential treatments for CMT1X — ... Read more
March 24, 2021March 24, 2021
Augustine Therapeutics Receives Funding to Research Treatments
News
Augustine Therapeutics has received €1.2 million (about $1.4 million) in funding for a research project that will seek to further ... Read more
March 23, 2021March 19, 2021
Pondering My Body’s Limitations Has Brought Me Back to Dancing
Columns, On My Own Two by Young Lee
A long time ago, a friend asked if there was anything I wished I could do that was outside my ... Read more
March 19, 2021March 19, 2021
#MDA2021 – Wearable Sensors Seen as Viable Measure of Function, Strength
News
A higher number of daily steps, measured through wearable sensors, was associated significantly with more lower-body strength and functional abilities ... Read more
March 17, 2021March 17, 2021
CMT2A Study Links Genetic Variants to Disease Severity
News
A large multicenter study on the natural history of Charcot-Marie-Tooth disease type 2A (CMT2A) correlates different genetic profiles and disease-causing ... Read more

April 14, 2021April 14, 2021

Gene Deficiency in CMTX May Limit Nerve Injury, Mouse Study Suggests

News

Sciatic motor nerves in mice lacking the GJB1 gene — defective in people with X-linked Charcot-Marie-Tooth (CMTX) disease — regenerated more after external injury than was seen in healthy mice, ... Read more

April 13, 2021April 9, 2021

Receiving an Offer of Help Reminded Me What It Means to Be a CMTer

Columns, On My Own Two by Young Lee

I had just exited the county building after getting my second shot of the COVID-19 vaccine and was making my way across the parking lot toward my car. Then I ... Read more

April 9, 2021April 9, 2021

$20,000 Grant Will Help Unveil How Certain Mutations Fuel CMT

News

Scientist Timothy Hines, PhD, was awarded a grant of $20,000 to establish cell models to study Charcot-Marie-Tooth (CMT) disease at the recent live-streamed Young Investigator Draft ceremony. The grant, co-funded ... Read more

April 7, 2021April 7, 2021

Pharnext Enrolls 1st Patient in PREMIER Trial Testing PXT3003 for CMT1A

News

The first patient has been enrolled in a pivotal Phase 3 trial testing Pharnext’s investigational therapy PXT3003 for Charcot-Marie-Tooth disease type 1A (CMT1A), the company announced. Called PREMIER (NCT04762758), the trial intends to recruit ... Read more

April 6, 2021April 2, 2021

Home Cooking Videos Showed a New Side of CMT Symptoms

Columns, On My Own Two by Young Lee

I’m no Gordon Ramsay. And it’d be insulting to actual professionals if I were to call myself a chef or even a home cook. However, I do feel fairly comfortable ... Read more

April 2, 2021April 2, 2021

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

News

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of ... Read more

March 31, 2021March 31, 2021

Interim Results of CMT&Me Study Presented in Webinar

News

Depression, hand-and-finger weakness, and a lengthy delay from symptoms to diagnosis contribute to the burdens felt by people living with Charcot-Marie-Tooth disease (CMT). Those are among the key findings of ... Read more

March 26, 2021March 26, 2021

CMTA Grants Researchers $1.1M to Advance Trials in CMT1X, CMT2A

News

The Charcot-Marie-Tooth Association (CMTA) has awarded $1.1 million to help scientists advance clinical trials evaluating potential treatments for CMT1X — a subtype comprising 15% of all cases among this group ... Read more

March 24, 2021March 24, 2021

Augustine Therapeutics Receives Funding to Research Treatments

News

Augustine Therapeutics has received €1.2 million (about $1.4 million) in funding for a research project that will seek to further characterize the biology of Charcot-Marie-Tooth disease (CMT), and to investigate ... Read more

March 23, 2021March 19, 2021

Pondering My Body’s Limitations Has Brought Me Back to Dancing

Columns, On My Own Two by Young Lee

A long time ago, a friend asked if there was anything I wished I could do that was outside my ability, capability, or means. The question wasn’t prompted by familiarity ... Read more