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CMTAreportSpring2023spreads
…CMTA Partner On ASO Treatment for CMT1A 2023 ‘Pivotal’ Year for PXT3003 Treatment For CMT1A as Manufacturing Shifts to U.S. CMTA partner Pharnext announced Jan. 26 that it has completed…
CMTAreportSummer2022web
…quite strong compared to the beginning of treatment. I always used a handrail going up and down stairs before treatment, but now I walk up and down with some pace…
CMTAfall2022web
…virtual meeting June 7. Mackenzie shared valuable information on treatment goals for bracing, treatment options, who’s a candi- date, what to expect at a visit and much more. The branch…
CMTAfall2022web
…virtual meeting June 7. Mackenzie shared valuable information on treatment goals for bracing, treatment options, who’s a candi- date, what to expect at a visit and much more. The branch…
2022.09.12_PREMIER-OLE_FPI_EN
…2022 has shown a good safety profile and continuous treatment effect of PXT3003 measured on the ONLS after 5 years of total treatment time. 123 Patients with mild-to-moderate CMT1A are…
CMTAreportSummer2022web
…quite strong compared to the beginning of treatment. I always used a handrail going up and down stairs before treatment, but now I walk up and down with some pace…
CMTA and ToolGen Announce CMT1A Collaboration
CMTA and ToolGen Announce Collaboration to Develop Potential Treatment for CMT1A Using Gene Therapy (Glenolden, PA, October 7, 2021) The Charcot–Marie–Tooth Association (CMTA), the largest philanthropic funder of CMT research…
Natural History of TRPV4 Neuromuscular Disease – CMT2C
…in animal models of this condition has shown that disease mutations cause TRPV4 to be overactive, and drugs that block the channel are effective as treatments in these models. This…
The Spring 2023 CMTA Report
…of interest: New CMT1X Mouse Model Patients as Partners in Research Tour of Conklin Lab Life-Changing Pharnext Moves Px3003 Production to Us ASO Treatment for CMT1A Circumnavigating the Globe New…
Innervator Hall of Fame
…invest in invaluable educational initiatives and treatment-focused research. Join our action-oriented game changers by becoming a CMTA INNERVATOR. Tracy Adamson Richard Arsenault Aaron Baker David Balbi Kelly Banner James Barker…