The European Medicines Agency Has Approved a Paediatric Investigation Plan for an Experimental Treatment of Charcot-Marie-Tooth Disease Type 1A
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The European Medicines Agency Has Approved a Paediatric Investigation Plan for an Experimental Treatment of Charcot-Marie-Tooth Disease Type 1A

The company Pharnext SA, which is developing the investigational drug PXT3003 for the treatment of Charcot-Marie-Tooth disease type 1A, has agreed upon a paediatric investigation plan for the drug with…

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Zebrafish Lab Research Could Produce Solutions for Neurodegenerative Disease Like CMT and MS
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Zebrafish Lab Research Could Produce Solutions for Neurodegenerative Disease Like CMT and MS

According to UVA Today, a University of Virginia professor, Sarah Kucenas, is studying glia cells in the brain of fish that could potentially help spear the way for the creation of…

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Gene Mutation Found in Family with CMT Helps Scientists Expand Knowledge on the Disease
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Gene Mutation Found in Family with CMT Helps Scientists Expand Knowledge on the Disease

According to Charcot-Marie-Tooth News, a Chinese family of five who all have the same type of Charcot-Marie-Tooth (CMT) disease share something else in common as well. Not only do they…

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Researchers Are Investigating a New Way to Monitor Disease Progression for People with Charcot-Marie-Tooth Disease Type 1
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Researchers Are Investigating a New Way to Monitor Disease Progression for People with Charcot-Marie-Tooth Disease Type 1

Researchers have found a new way to monitor disease progression in people with Charcot-Marie-Tooth disease type 1 (CMT1), reports Charcot Marie Tooth News. CMT1 is an inherited genetic condition that…

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An Experimental Treatment for FSHD Has Been Awarded Fast Track Designation by the FDA
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An Experimental Treatment for FSHD Has Been Awarded Fast Track Designation by the FDA

Acceleron Pharma has just received Fast Track designation from the US Food and Drug Administration (FDA) for their experimental treatment ACE-083 designed to treat patients with facioscapulohumeral muscular dystrophy (FSHD).…

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Voice Technology and Cryptocurrency Changing Charcot-Marie-Tooth Disease Research
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Voice Technology and Cryptocurrency Changing Charcot-Marie-Tooth Disease Research

Collaboration often leads to greater success. Two advocacy groups, the Hereditary Neuropathy Foundation and True Reply have done just that to benefit patients with Charcot-Marie-Tooth disease. Their combined efforts focus…

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Genetic Testing is Necessary to Avoid Nerve-Damaging Chemotherapy for Patients With Charcot-Marie-Tooth Disease
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Genetic Testing is Necessary to Avoid Nerve-Damaging Chemotherapy for Patients With Charcot-Marie-Tooth Disease

A story from Charcot-Marie-Tooth News highlights the necessity for genetic testing and a physical examination prior to chemotherapy in order to prevent nerve damage in people with asymptomatic Charcot-Marie-Tooth disease…

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Understanding The Mechanisms of Charcot-Marie-Tooth Disease Can Help Develop Treatments
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Understanding The Mechanisms of Charcot-Marie-Tooth Disease Can Help Develop Treatments

According to a story from news-medical.net, researchers from The Scripps Research Institute have successfully outlined a new path for the treatment of Charcot-Marie-Tooth disease subtype 2D (CMT2D). The scientists hope…

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Copper Deficiency a Possible Culprit in Charcot-Marie-Tooth Disease
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Copper Deficiency a Possible Culprit in Charcot-Marie-Tooth Disease

According to a recent study, low copper levels could have a hand in leading to Charcot-Marie-Tooth disease, Charcot-Marie-Tooth News reports. Charcot-Marie-Tooth disease (CMT) is a neurological disorder that affects the…

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7-Year-Old with Charcot-Marie-Tooth Nears Fundraising Goal That Would Let Him Walk Again
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7-Year-Old with Charcot-Marie-Tooth Nears Fundraising Goal That Would Let Him Walk Again

Just like any other 7-year-old little boy, Ethan Perkins has big dreams. He loves trucks and reading, and wants to study fossils as a paleontologist reported KXLY. He also wants to be…

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Editor’s Choice: Staying Strong, Fighting for Treatment, and Feeling Vulnerable- Just Another Day in the Life of Rare Patients

Happy Father's Day Weekend Patient Worthians! This week we have an amazing essay written by CMT Warrior Sarah Magno regarding her personal experience overcoming rare disease obstacles. We also have some…

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Rare Patient Spotlight: Young Woman Stays Strong in the Face of CMT
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Rare Patient Spotlight: Young Woman Stays Strong in the Face of CMT

In collaboration with the Hereditary Neuropathy Foundation (HNF), Patient Worthy asked members of the Charcot-Marie Tooth (CMT) community to tell us their stories, in hopes of inspiring everyone in the rare community…

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Editor’s Choice: Rare Adolescence, Rare Parenting and Rare… CEOing

Happy Memorial Day Weekend Patient Worthians! This week we have an inspiring interview with the CEO and Founder of the Hereditary Neuropathy Foundation. We also have a motivating story on a cheerleader battling Friedreich's…

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Cancer Survivor, Chronic Illness Patient, CEO, and Mom: How Allison Balances a Busy Life
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Cancer Survivor, Chronic Illness Patient, CEO, and Mom: How Allison Balances a Busy Life

Moms: The ultimate multitaskers. Whether they're stay-at-home moms or working mom, these strong ladies have a way of getting stuff done, come hell or high water - or a chronic…

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