Updates

May 28, 2020May 28, 2020
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays ... Read more
May 26, 2020May 26, 2020
For CMTers Like Me, It’s Important to ‘Just Keep Moving’

“Just keep moving.” For Steve O’Donnell, a Charcot-Marie-Tooth Association board member and founder of the nonprofit Therapies for Inherited Neuropathies, those are words to live by. ... Read more
May 25, 2020May 25, 2020
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 ... Read more
May 22, 2020May 22, 2020
New MCM3AP Gene Mutation Linked to CMT in Chinese Girl

A newly identified mutation in the MCM3AP gene caused Charcot-Marie-Tooth disease (CMT) in a girl with early motor development delay, a study reports. Findings ... Read more
May 20, 2020
Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together ... 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

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

For CMTers Like Me, It’s Important to ‘Just Keep Moving’

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

New MCM3AP Gene Mutation Linked to CMT in Chinese Girl

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

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It’s Time to Be Gentler with Myself

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

CMT2 Subtypes Lead to Common Nerve Cell and Muscle Deficits, Worm Study Suggests

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Gaming Shouldn’t Only Benefit the Able-bodied

Yeast Model May Help Assess CMT4A Mutations

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Finding Comfort and Community in Gaming

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

How Charcot-Marie-Tooth Is Helping Me Cope with the Pandemic

Extended Protein Form Linked with Myelin Damage, May Underlie CMT, Mouse Study Finds

Lessons from Bernadette to Help Us During the Quarantine

Nerve Conduction Changes Not Linked to Quality of Life and Hand Function in CMT1A Patients, Study Says

NORD’s Advice on COVID-19: You’re Stronger Than You Think

With $30M Investment, Regenacy Plans Phase 2 Trial Testing Ricolinostat for CMT Type 2

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

PXT3003 Designated Promising Innovative Medicine in UK for CMT1A Treatment

Like Many, My Feelings About the COVID-19 Craziness Are Complicated

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

Pharnext Raises 7.7M Euros to Develop PXT3003 Therapy for CMT1A

Mutation in SEPT9 Gene Can Lead to CMT, Report Says

It’s Time for Disabled Narratives to Take the Spotlight

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

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COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

For CMTers Like Me, It’s Important to ‘Just Keep Moving’

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

New MCM3AP Gene Mutation Linked to CMT in Chinese Girl

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Subscribe to our mailing list

It’s Time to Be Gentler with Myself

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

CMT2 Subtypes Lead to Common Nerve Cell and Muscle Deficits, Worm Study Suggests

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Gaming Shouldn’t Only Benefit the Able-bodied

Yeast Model May Help Assess CMT4A Mutations

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Finding Comfort and Community in Gaming

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

How Charcot-Marie-Tooth Is Helping Me Cope with the Pandemic

Extended Protein Form Linked with Myelin Damage, May Underlie CMT, Mouse Study Finds

Lessons from Bernadette to Help Us During the Quarantine

Nerve Conduction Changes Not Linked to Quality of Life and Hand Function in CMT1A Patients, Study Says

NORD’s Advice on COVID-19: You’re Stronger Than You Think

With $30M Investment, Regenacy Plans Phase 2 Trial Testing Ricolinostat for CMT Type 2

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

PXT3003 Designated Promising Innovative Medicine in UK for CMT1A Treatment

Like Many, My Feelings About the COVID-19 Craziness Are Complicated

Managing My Anxiety During the COVID-19 Pandemic

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

Pharnext Raises 7.7M Euros to Develop PXT3003 Therapy for CMT1A

Mutation in SEPT9 Gene Can Lead to CMT, Report Says

It’s Time for Disabled Narratives to Take the Spotlight

Pin It on Pinterest