Updates

August 7, 2020August 7, 2020
$10,000 Community Grant Supports CMT Australia Youth Efforts

CMT Australia, a charity association aiming to raise awareness and empower people with Charcot-Marie-Tooth disease (CMT) to live their lives to the fullest, has ... Read more
August 4, 2020July 31, 2020
Trying to Better Manage the Little Frustrations

I was manning three active burners on the stovetop, deep into my food preparation for the week. On one of the front burners was ... Read more
August 4, 2020August 4, 2020
PXT3003’s Components Work in Synergy to Improve Outcomes in CMT1A, Study Finds

The three components of the investigational therapy PXT3003 are all essential to significantly improve features of Charcot-Marie-Tooth disease (CMT) type 1A in cell and animal ... Read more
July 31, 2020July 31, 2020
Scientists Find Way to Create Models of CMT Motor Neurons for Research

A new protocol allows researchers to generate spinal motor neurons derived from Charcot-Marie-Tooth disease (CMT) patients, creating useful models for better understanding the disease ... Read more
July 29, 2020July 29, 2020
Phase 1/2a Trial of Gene Therapy Engensis for CMT1A Begins in Korea

Helixmith has launched a Phase 1/2 clinical trial in South Korea to investigate its gene therapy product Engensis (VM202) for the treatment of muscle ... Read more

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September 22, 2017September 22, 2017

Despite Functional Limitations, Few CMT Patients Receive Physical and Occupational Therapy

News

Most people with Charcot-Marie-Tooth (CMT) disease do not receive physical or occupational therapy, despite having extensive problems with physical functioning that limit mobility and quality of life, according to a report presented at the American Association of Neuromuscular & ... Read more

Updates

$10,000 Community Grant Supports CMT Australia Youth Efforts

Trying to Better Manage the Little Frustrations

PXT3003’s Components Work in Synergy to Improve Outcomes in CMT1A, Study Finds

Scientists Find Way to Create Models of CMT Motor Neurons for Research

Phase 1/2a Trial of Gene Therapy Engensis for CMT1A Begins in Korea

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Letting Go of My Pride and Asking for Help

Shark Tooth Cycling Opens as Nonprofit, Encouraging Active Life With CMT

No, You Aren’t a ‘Savage’ for Being Unable to Return That Cart

Study Sheds New Light on How HARS1 Mutations Cause CMT in Cells, Zebrafish

Chemo-induced Nerve Damage Less Likely in Certain CMT Patients, Study Finds

Telehealth Helped in Therapy Sessions With CMTX Patient Suspected of COVID-19

A Lesson from Woody for When I Feel Down

Common Mutations of CMT4 Subtypes Not Found in Iranian Patients, Study Reports

DTx Pharma Study Discovers Multiple Therapeutic Candidates for CMT1A

Problems with Mitochondria of Neurons May Underlie CMTX6, Study Finds

I Take Offense to the Ableist Jokes About President Trump

mTORC1 Overactivation Linked to CMT4B Myelin Defects, Early Study Shows

Tips for Photography Enthusiasts with Disabilities

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

Hearing Difficulties in CMT Linked to Nerve Signaling Problems in Study

Pharnext Plans Final Trial to Support PXT3003 Approval for CMT1A

Copy Number Variations Helpful in Spotting Potential CMT Mutations

I’m Trying Not to Let Fear Limit Me

Changes in Sciatic Nerve of Thigh May Reflect Disability in CMT1a Patients

Coalition Will Address Racial Disparities in Rare Disease Communities

Disease in Family Traced to 2 Separate Mutations Working ‘in Tandem’

I’m Trying to Make Exercise Irresistible

New Mutation in PMP22 Gene Linked to Severe, Early-onset Form of CMT1A

Pregnancy, Delivery Complications Not Higher in Women With CMT, Study Reports

Despite Functional Limitations, Few CMT Patients Receive Physical and Occupational Therapy

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$10,000 Community Grant Supports CMT Australia Youth Efforts

Trying to Better Manage the Little Frustrations

PXT3003’s Components Work in Synergy to Improve Outcomes in CMT1A, Study Finds

Scientists Find Way to Create Models of CMT Motor Neurons for Research

Phase 1/2a Trial of Gene Therapy Engensis for CMT1A Begins in Korea

Subscribe to our mailing list

Letting Go of My Pride and Asking for Help

Shark Tooth Cycling Opens as Nonprofit, Encouraging Active Life With CMT

No, You Aren’t a ‘Savage’ for Being Unable to Return That Cart

Study Sheds New Light on How HARS1 Mutations Cause CMT in Cells, Zebrafish

Chemo-induced Nerve Damage Less Likely in Certain CMT Patients, Study Finds

Telehealth Helped in Therapy Sessions With CMTX Patient Suspected of COVID-19

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

A Lesson from Woody for When I Feel Down

Common Mutations of CMT4 Subtypes Not Found in Iranian Patients, Study Reports

DTx Pharma Study Discovers Multiple Therapeutic Candidates for CMT1A

Problems with Mitochondria of Neurons May Underlie CMTX6, Study Finds

I Take Offense to the Ableist Jokes About President Trump

mTORC1 Overactivation Linked to CMT4B Myelin Defects, Early Study Shows

Tips for Photography Enthusiasts with Disabilities

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

Hearing Difficulties in CMT Linked to Nerve Signaling Problems in Study

Pharnext Plans Final Trial to Support PXT3003 Approval for CMT1A

Copy Number Variations Helpful in Spotting Potential CMT Mutations

I’m Trying Not to Let Fear Limit Me

Changes in Sciatic Nerve of Thigh May Reflect Disability in CMT1a Patients

Coalition Will Address Racial Disparities in Rare Disease Communities

Disease in Family Traced to 2 Separate Mutations Working ‘in Tandem’

I’m Trying to Make Exercise Irresistible

New Mutation in PMP22 Gene Linked to Severe, Early-onset Form of CMT1A

Pregnancy, Delivery Complications Not Higher in Women With CMT, Study Reports

Pin It on Pinterest