Author Archives: Margarida Azevedo, MSc

Pharnext recently provided an update from its ongoing Phase 3 clinical trials evaluating PXT3003 for treating Charcot-Marie-Tooth disease type 1A (CMT1A) in adults, and also announced that some top-line results are expected by October 2018. PXT3003 is a fixed-dose combination of baclofen, naltrexone and sorbitol with orphan…

Grünenthal, the Lead Discovery Center (LDC) and the Max-Planck Society have signed a research collaboration agreement to develop new therapies for Charcot-Marie-Tooth type 1A (CMT1A). Two branches of Max-Planck are involved: Max-Planck Innovation and the Max Planck Institute of Experimental Medicine (MPI-EM). These two will work with…

The French biopharmaceutical company Pharnext recently announced that its candidate for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A) was granted priority review by the China Food and Drug Administration (CFDA). PXT3003 is a fixed-dose combination of baclofen, naltrexone, and sorbitol. The therapy candidate has received orphan drug status in both…

Like many of us, people living with Charcot-Marie-Tooth (CMT) disease make resolutions to become healthier and more fit. But while starting something new can be exciting, finding out what exercises are best when you have a limiting condition can be tough to navigate. In the Exercise and Physical Therapy area…

True Reply recently committed to donating up to $200,000 from its cryptocurrency sale to support the Hereditary Neuropathy Foundation (HNF) and the Charcot-Marie-Tooth (CMT) disease community. The donation is part of an ongoing collaboration between the tech startup and the nonprofit foundation aimed at enhancing patient-focused…

The online community GeneFo will hold a free webinar this month to provide those living with Charcot-Marie-Tooth (CMT) disease with updated insights into exercise and physical therapy strategies to help manage symptoms. The event, titled “Exercise and Physical Therapy Strategies for CMT Symptoms,” will be held March 29…

The heartwarming story of students from three Massachusetts high schools banding together to help raise money to find a cure for the extremely rare neuropathy Charcot Marie Tooth, Type 4J (CMT4J) was featured in the Boston Globe recently. Students from Hamilton-Wenham, Ipswich, and Danvers high schools…

The connection between artist Andrew Wyeth’s most famous painting, Christina’s World, and Charcot-Marie-Tooth (CMT), a group of inherited disorders of the peripheral nervous system, will unfold as the Charcot-Marie-Tooth Association (CMTA) hosts its 8th Annual CMTA Gala to raise funds for clinical trials of…

Flex Pharma has initiated its Phase 2 COMMIT trial to evaluate the drug candidate FLX-787 in patients with Charcot-Marie-Tooth (CMT) disease who have debilitating muscle cramps. The COMMIT study (NCT03254199) is a randomized, double-blind, trial that will be conducted at 20 centers the U.S. under Flex Pharma’s open investigational…

Cure CMT4J, a nonprofit foundation started by a  family whose daughter was diagnosed with Charcot Marie Tooth Type 4, or CMT4J, several years ago, is holding a fundraising gala in Newburyport, Massachusetts, this month to help advance a clinical trial. “When we received Talia’s…