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…
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 a story from Charcot-Marie-Tooth News, a recent study has demonstrated the potential impacts of an RNA-based therapy as a treatment for type 1A Charcot-Marie-Tooth disease. The treatment was…
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…
Pharnext has released an update on the clinical development of PXT3003, a treatment for Charcot-Marie-Tooth disease type 1A (CMT1A). According to the biopharmaceutical company, they are on track to dose…
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…
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