July 1, 2020July 1, 2020
DTx Pharma Study Discovers Multiple Therapeutic Candidates for CMT1A
News
DTx Pharma has discovered new molecules that could be treatment candidates for Charcot-Marie-Tooth disease (CMT) type 1A (CMT1A), reaching ... Read more
June 26, 2020June 26, 2020
Problems with Mitochondria of Neurons May Underlie CMTX6, Study Finds
News
Problems with energy production and mitochondria, the powerhouses of cells, may be the underlying cause of an X-linked form ... Read more
June 23, 2020June 19, 2020
I Take Offense to the Ableist Jokes About President Trump
Columns, On My Own Two by Young Lee
“TrumpWearsAdultDiapers”: It’s a hashtag I first noticed trending in the United States on the morning of June 14. And ... Read more
June 23, 2020June 23, 2020
mTORC1 Overactivation Linked to CMT4B Myelin Defects, Early Study Shows
News
Mutations that cause Charcot-Marie-Tooth disease type 4B (CMT4B) lead to an increase in the activity of a protein called ... Read more
June 22, 2020June 19, 2020
Tips for Photography Enthusiasts with Disabilities
CMT and Me - a Column by Jill Price, Columns
Recently, I joined a photography group for moms, and with them, I’ve been learning new techniques to feed my ... Read more
June 22, 2020June 22, 2020
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic
News, syndicated
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during ... Read more
June 19, 2020June 19, 2020
Hearing Difficulties in CMT Linked to Nerve Signaling Problems in Study
News
People with Charcot-Marie-Tooth disease (CMT) may have difficulties in understanding speech, even in the absence of hearing loss, caused ... Read more
June 16, 2020June 16, 2020
Pharnext Plans Final Trial to Support PXT3003 Approval for CMT1A
News
With guidance from the U.S. Food and Drug Administration (FDA), Pharnext is planning a pivotal, final Phase 3 clinical ... Read more
June 12, 2020June 12, 2020
Copy Number Variations Helpful in Spotting Potential CMT Mutations
News
Testing for large deletions or duplications in certain DNA regions, a form of mutation called copy number variations, is ... Read more
June 9, 2020June 5, 2020
I’m Trying Not to Let Fear Limit Me
Columns, On My Own Two by Young Lee
The indoor track of my alma mater’s gym smells like rubber and sweat. It has a specific warmth to ... Read more
June 9, 2020June 9, 2020
Changes in Sciatic Nerve of Thigh May Reflect Disability in CMT1a Patients
News
Changes to nerve tissue in the thigh appear to relate to clinical disability in people with Charcot-Marie-Tooth disease type ... Read more
June 9, 2020June 10, 2020
Coalition Will Address Racial Disparities in Rare Disease Communities
News
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in ... Read more

July 1, 2020July 1, 2020

DTx Pharma Study Discovers Multiple Therapeutic Candidates for CMT1A

News

DTx Pharma has discovered new molecules that could be treatment candidates for Charcot-Marie-Tooth disease (CMT) type 1A (CMT1A), reaching the second milestone of a study designed to find molecules ... Read more

June 26, 2020June 26, 2020

Problems with Mitochondria of Neurons May Underlie CMTX6, Study Finds

News

Problems with energy production and mitochondria, the powerhouses of cells, may be the underlying cause of an X-linked form of Charcot-Marie-Tooth disease (CMT) called CMTX6, a study in patient-derived ... Read more

June 23, 2020June 19, 2020

I Take Offense to the Ableist Jokes About President Trump

Columns, On My Own Two by Young Lee

“TrumpWearsAdultDiapers”: It’s a hashtag I first noticed trending in the United States on the morning of June 14. And I remember looking at it with mixed emotions. On one ... Read more

June 23, 2020June 23, 2020

mTORC1 Overactivation Linked to CMT4B Myelin Defects, Early Study Shows

News

Mutations that cause Charcot-Marie-Tooth disease type 4B (CMT4B) lead to an increase in the activity of a protein called mTORC1 that is responsible for the problems with myelin that characterize ... Read more

June 22, 2020June 19, 2020

Tips for Photography Enthusiasts with Disabilities

CMT and Me - a Column by Jill Price, Columns

Recently, I joined a photography group for moms, and with them, I’ve been learning new techniques to feed my passion. However, there are times that I find these techniques ... Read more

June 22, 2020June 22, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

News, syndicated

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will ... Read more

June 19, 2020June 19, 2020

Hearing Difficulties in CMT Linked to Nerve Signaling Problems in Study

News

People with Charcot-Marie-Tooth disease (CMT) may have difficulties in understanding speech, even in the absence of hearing loss, caused by problems with the nerve cells that carry signals from ... Read more

June 16, 2020June 16, 2020

Pharnext Plans Final Trial to Support PXT3003 Approval for CMT1A

News

With guidance from the U.S. Food and Drug Administration (FDA), Pharnext is planning a pivotal, final Phase 3 clinical trial of its investigational therapy PXT3003 for the treatment of ... Read more

June 12, 2020June 12, 2020

Copy Number Variations Helpful in Spotting Potential CMT Mutations

News

Testing for large deletions or duplications in certain DNA regions, a form of mutation called copy number variations, is important in identifying new mutations that cause Charcot-Marie-Tooth disease (CMT), ... Read more

June 9, 2020June 5, 2020

I’m Trying Not to Let Fear Limit Me

Columns, On My Own Two by Young Lee

The indoor track of my alma mater’s gym smells like rubber and sweat. It has a specific warmth to it. The repeated impacts of dozens of feet send shockwaves ... Read more