Charcot-Marie-Tooth disease – Page 6

October 2018 is the Expected Date for Top-Line Results From a Phase 3 Trial of an Experimental Treatment for Charcot-Marie-Tooth Disease

Post author:Anna Hewitt
Post published:July 27, 2018
Post category:Charcot-Marie-Tooth disease Rare Disease

An ongoing Phase 3 clinical trial program is investigating the drug PXT3003 as an experimental treatment for Charcot-Marie-Tooth disease type 1A in adults. Pharnext SA says that they expect to…

Source: Pixabay

What’s the Best Way to Meaure Charcot-Marie-Tooth Disease Outcomes?

Post author:Dawn Scott
Post published:July 16, 2018
Post category:Charcot-Marie-Tooth disease Rare Disease

Recently in Charcot‐Marie‐Tooth news, progress has been made in identifying better testing to use as outcome measures for people living with Charcot-Marie-Tooth disease type 1A. A study called “Outcome measures…

Source: Pixabay

MDA Grants $1,000,000 for Charcot-Marie-Tooth Disease Research

Post author:Samuel Sachs
Post published:July 16, 2018
Post category:Charcot-Marie-Tooth disease Muscular Dystrophy

According to a July 9 report by the Muscular Dystrophy Association (MDA), the MDA awarded a large grant to researchers studying Charcot-Marie-Tooth disease. The grant, totaling over $1,000,000, was awarded…

stevepb / Pixabay

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

Post author:Anna Hewitt
Post published:July 16, 2018
Post category:Charcot-Marie-Tooth disease

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…

Source: Pixabay

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

Post author:Athena Lee
Post published:June 18, 2018
Post category:Charcot-Marie-Tooth disease Multiple Sclerosis Muscular Dystrophy

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…

qimono / Pixabay

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

Post author:Athena Lee
Post published:June 13, 2018
Post category:Charcot-Marie-Tooth disease Rare 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…

Source: Pixabay

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

Post author:Anna Hewitt
Post published:May 24, 2018
Post category:Charcot-Marie-Tooth disease

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…

source: pixabay.com

Learn a Little About Neuropathy During National Neuropathy Awareness Week

Post author:Jean Martell
Post published:May 15, 2018
Post category:Charcot-Marie-Tooth disease Rare Disease Timely

May 12th through May 16th is National Neuropathy Awareness Week! It is reported that about 1 in 15 Americans are afflicted by neuropathy - but many of them do not…

RobinHiggins / Pixabay

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

Post author:Anna Hewitt
Post published:May 3, 2018
Post category:Charcot-Marie-Tooth disease Muscular Dystrophy

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).…

Source: Pixabay

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

Post author:Samuel Sachs
Post published:April 16, 2018
Post category:Charcot-Marie-Tooth disease

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…

source: pixabay.com

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

Post author:James Moore
Post published:April 5, 2018
Post category:Charcot-Marie-Tooth disease Rare 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…

jarmoluk / Pixabay

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

Post author:James Moore
Post published:March 21, 2018
Post category:Charcot-Marie-Tooth disease

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…

Source: Pixabay

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

Post author:Katie Campbell
Post published:March 19, 2018
Post category:Charcot-Marie-Tooth disease Rare 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…

Source: Pixabay

New Study Identifies Compounds that may Treat Mutations in Charcot-Marie-Tooth Disease

Post author:Samuel Sachs
Post published:January 12, 2018
Post category:Charcot-Marie-Tooth disease

A new study uncovered two proteins that may be able to reduce symptoms in certain diseases. The proteins may be especially effective at treating diseases with nonsense mutations, such as…

Source: Pixabay

Here’s How You Can Help the Charcot-Marie-Tooth Association!

Post author:Patient Worthy Contributor
Post published:January 11, 2018
Post category:Charcot-Marie-Tooth disease

Patient Worthy recieved a note from CMT Warrior and Patient Worthy Contributor Quentin Martin of a worthwhile cause, that we think all our readers should know about! Quentin writes: If…

Source: Pixabay

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

Post author:Meagan Fulps
Post published:December 27, 2017
Post category:Charcot-Marie-Tooth disease

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…

source: pixabay.com

The Sleep Aid That May Help Patients with Charcot-Marie-Tooth Disease

Post author:Patient Worthy Contributor
Post published:November 7, 2017
Post category:Charcot-Marie-Tooth disease

At some point in your life, you might have taken a melatonin supplement from a bottle to get to sleep, or to get over jet-lag. Your body naturally produces melatonin…

Source: Pixabay

Robotics Students Give Rare Disease Girl a ‘Hand’

Post author:Andres Rovira
Post published:November 7, 2017
Post category:Charcot-Marie-Tooth disease

Eleven-year-old Talia Duff finds herself in the rare situation of being one of only 22 people in the world to suffer from a rare disease. Last year, Jocelyn Duff received…

CMT and Experiences at the Academy for the Blind

Post author:Patient Worthy Contributor
Post published:September 26, 2017
Post category:Charcot-Marie-Tooth disease Rare Disease

The first story that comes to Quentin's mind about a turning point in his life is about his attendance at a school for the blind. When Quentin lost his vision…

My Challenging, Yet Rewarding Life, With Charcot-Marie-Tooth Disease

Post author:Patient Worthy Contributor
Post published:June 22, 2017
Post category:Charcot-Marie-Tooth disease

In collaboration with the Hereditary Neuropathy Foundation (HNF), Patient Worthy held an essay contest for tickets to a Charcot-Marie-Tooth disease event. We asked members of the Charcot-Marie Tooth (CMT) community to tell…

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

Post author:Patient Worthy Contributor
Post published:June 16, 2017
Post category:Charcot-Marie-Tooth disease Cystic Fibrosis Mucolipidosis Type III Rare Disease Tardive Dyskinesia

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…

Sarah M kayaking.

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

Post author:Patient Worthy Contributor
Post published:June 12, 2017
Post category:Charcot-Marie-Tooth disease

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…

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

Post author:Patient Worthy Contributor
Post published:May 26, 2017
Post category:Charcot-Marie-Tooth disease Friedreich ataxia Rare Disease

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…

Allison. Being an all-star.

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

Post author:Jean Martell
Post published:May 19, 2017
Post category:Charcot-Marie-Tooth disease Rare Disease

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…

October 2018 is the Expected Date for Top-Line Results From a Phase 3 Trial of an Experimental Treatment for Charcot-Marie-Tooth Disease

What’s the Best Way to Meaure Charcot-Marie-Tooth Disease Outcomes?

MDA Grants $1,000,000 for Charcot-Marie-Tooth Disease Research

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

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

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

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

Learn a Little About Neuropathy During National Neuropathy Awareness Week

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

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

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

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

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

New Study Identifies Compounds that may Treat Mutations in Charcot-Marie-Tooth Disease

Here’s How You Can Help the Charcot-Marie-Tooth Association!

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

The Sleep Aid That May Help Patients with Charcot-Marie-Tooth Disease

Robotics Students Give Rare Disease Girl a ‘Hand’

CMT and Experiences at the Academy for the Blind

My Challenging, Yet Rewarding Life, With Charcot-Marie-Tooth Disease

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

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

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

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

Metastatic Breast Cancer: Navigating Grief

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The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

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