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…
Charcot-Marie-Tooth disease (CMT) is associated with a number of genetic mutations. In fact, the different mutations signify specific CMT subtypes, such as CMT1 or CMT3. According to Charcot-Marie-Tooth News,…
Over the next 2-2.5 years, biotech spin-out company Augustine Therapeutics will be working to understand both the underlying biology of Charcot-Marie-Tooth disease (CMT) and potential therapeutic options for patients.…
Charcot-Marie Tooth disease (CMT) may be the most common inherited disorder of the peripheral nervous system, but that only puts the official patient count in Denmark at about 22.5 cases…
A Phase 3 clinical trial run by Pharnext for Charcot-Marie-Tooth disease Type 1A (CMT1A) has just been awarded 13.3 million dollars. $7.2 million comes from existing shareholders and $6 million comes…
According to Charcot-Marie-Tooth News, the CMT Research Foundation has announced that they will provide additional funding for the development of a gene therapy for Charcot-Marie-Tooth disease Type 1 (CMT1A). The…
James Dahlman, a scientist and professor at both Emory University and the Georgia Institute of Technology, has partnered with the CMT Research Foundation. The aim of this partnership is to…
A recent study has illuminated the need for further research into how different forms of exercise may provide certain benefits for patients living with Charcot-Marie-Tooth disease (CMT). This study was…
Pharnext has announced that they will soon open enrollment for their PREMIER trial of PXT3003, a Charcot-Marie-Tooth disease type 1A (CMT1A) treatment. Beginning in March, researchers will search for 350…
In a recent study, researchers have uncovered a second gene located within the mitochondria that is associated with Charcot-Marie-Tooth Disease (CMT). This study was recently published in Annals of Neurology. CMT…
A woman living with Charcot-Marie-Tooth Disease type 2 (CMT2) has experienced symptom improvement following therapy with an anti-inflammatory compound found in green tea. This study was recently published in Medicina.…
According to Charcot-Marie-Tooth News, researchers have discovered a new subtype of Charcot-Marie-Tooth disease (CMT) linked to the sorbitol dehydrogenase (SORD) gene. Read the full study findings published in Nature Genetics. SORD Gene…
As reported in Charcot Marie Tooth News; researchers have devised a new measure of quality of life for youth with Charcot-Marie-Tooth disease. While of course, researchers could assess the youth’s…
What are neuromuscular junctions and how do they play a role in the development of Charcot-Marie-Tooth disease (CMT)? According to Charcot-Marie-Tooth News, researchers discovered that structural neuromuscular junction changes caused…
Medical professionals have known for a long time that peripheral nerve cell damage plays a major role in Charcot-Marie-Tooth (CMT) disease. A recent study published in Human Molecular Genetics has suggested that…
If you have a rare condition and are also looking to start a family, you might question: how will my condition impact my pregnancy? Will I be able to have…
Patient input is extremely important when evaluating treatments for rare diseases; Charcot-Marie-Tooth disease (CMT) is no exception. In order to understand the impact of different CMT therapies, researchers from the…
According to a story from Charcot-Marie-Tooth News, a recent cell-based study has revealed that Charcot-Marie-Tooth disease type 1C and type 4J share a common pathway malfunction. This is despite the…
According to a story in Charcot-Marie-Tooth News, the CMT&Me study has launched a competition in Europe with the purpose of raising awareness and money for CMT and related disorders. Any…
This week Hallmark Movies and Mysteries premiered The Christmas Bow, which features Joy Perry, a woman with Charcot-Marie-Tooth disease, playing a woman with CMT. And while CMT has been known…
According to a story from Charcot-Marie-Tooth News, patients living with Charcot-Marie-Tooth disease have reported in a recent survey that their symptoms of pain and decline in mobility increased during the…
Medical professionals are looking towards patient-reported outcomes more and more as a source for research. In fact, a study titled "Patient reported impact of Charcot-Marie-Tooth disease: protocol for a real…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
