Written by Lily Sander By the time I was age four, I started experiencing unexplained symptoms such as severely turned in feet, which caused extreme pain and mobility issues. Terrified,…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Written by Peter de Silva As a young person, I tripped and fell on a regular basis. Including during one little league baseball game which we might have won -…
Imagine being Rowan Marshall, a seven-year-old first-grade student with a debilitating rare disease. Rowan made it through the new student formalities. He is doing well at school. Now he faces…
Jenny Decker’s disease, Charcot-Marie-Tooth (CMT), is named after the three doctors who first discovered the disease in 1886. CMT is a progressive neurological disease that affects approximately 2.6…
Jenny Decker of Fenton, MO. interviewed with KMOV4 explaining that she was unable to walk when she was three years old due to a rare disease called Charcot-Marie-Tooth disease. Doctors…
According to a story published on Charcot-Marie-Tooth News, a recent real-world study highlighted the impacts and symptoms of Charcot-Marie-Tooth (CMT) disease type 1A. The most common form of the illness,…
The loss of axons (nervous system wiring) has been responsible for various neurodegenerative diseases such as peripheral neuropathies, Parkinson’s disease, glaucoma, and amyotrophic lateral sclerosis (ALS). According to a…
For as long as he could remember, Georgie Davey had one word that he thought described himself: “clumsy.” When he was a child, Georgie would frequently trip and fall. He…
Happy Friday! The days are getting shorter and the first cooling winds of autumn have started to blow. This week, we have stories on Charcot-Marie-Tooth Disease Awareness Month, a special…
If you are interested in learning about CMT or are even a little curious, this article will help to shed some light on this rare disease. You are invited by…
The Phase 3 PLEO-CMT clinical trial evaluated the safety, efficacy, and tolerability of PXT3003 for patients with Charcot-Marie-Tooth disease type 1A (CMT1A). Altogether, 323 people enrolled. During the trial, researchers…
According to a story from Charcot-Marie-Tooth News, the Global CMT Research Convention is set to take place in Cambridge, Massachusetts on September 16-17, 2022. Organized by the CMT Research Foundation,…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to a story from Charcot-Marie-Tooth News, a recent study using a mouse model of Charcot-Marie-Tooth disease type 1A produced some interesting results. The researchers found that administration of theophylline…
According to Charcot-Marie-Tooth News, researchers have associated HINT1 gene mutations with neuropathy in Greek patients with Charcot-Marie-Tooth disease (CMT). In the past, HINT1 was linked to CMT in central and southeastern Europe. However, researchers…
Although Jackson, age 62, has had CMT for ten years, he only divulged his illness this month. According to an article on eathis.com, Jackson chose a recently aired segment of…
Alan Jackson has been a big part of the country music scene for a long time, with songs like Remember When and Livin' On Love. Recently, he revealed to his…
It's no secret that genetic mutations can alter the way our bodies - and bodily processes - work. For example, explains Charcot-Marie-Tooth News, GARS gene mutations stop protein production in ribosomes. These…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
September is Charcot-Marie-Tooth disease (CMT) Awareness Month! The Charcot-Marie-Tooth Disease Association has put together 5 different ways you can help spread awareness of this rare disease this month. The Association…
In medical research, researchers often use a variety of non-human models to expand their understanding of various diseases or potential treatments. For example, mice models, or non-human primates, are frequently…
In case you missed it - a news release from biopharmaceutical company Pharnext SA ("Pharnext") shared that on July 12, 2021, the first European patient enrolled in the Phase 3…
A recent study has examined what risk factors exist for patients diagnosed with myotonic dystrophy, the most common of all muscular dystrophies. This is a progressive, hereditary condition. Regarding cardiac…
Partnerships within the medical community play a huge role in advancing research and developing treatments. According to Charcot-Marie-Tooth News, a new partnership has formed between Dr. Alessandra Bolino, PhD, the…
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