Earlier this year, I interviewed Dr. Arthur Suckow, the CEO and co-founder of DTx Pharma, about the innovative RNA therapeutics being developed for Charcot-Marie-Tooth disease type 1A (CMT1A). In…
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 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…
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…
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 a story from Charcot-Marie-Tooth News, the drug company DTx Pharma has recently obtained critical funding that will go towards the development of new RNA-based treatments for Charcot-Marie-Tooth disease…
According to a story from Charcot-Marie-Tooth News, an experimental HDAC6 inhibitor called CKD-504 appeared to restore myelination in mouse and cell models of Charcot-Marie-Tooth disease type 1A in a recent…
According to a story from Charcot-Marie-Tooth News, a recent study suggests that the three components of the investigational therapy PXT3003 work in synergy to improve the condition of patients with…
A study conducted by researchers from Aix-Marseille University in France has revealed a new way to track disease progression of Charcot-Marie-Tooth disease type 1a (CMT1a)- changes to the nerves within the thigh.…
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…
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