According to a story from Charcot-Marie-Tooth News, a recent study suggests that changes in the levels of two proteins in the body could serve as biomarkers to indicate the progression…
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…
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…
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…
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…
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…
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…
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…
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…
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).…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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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