New Study Measures Progression of CMT1A Nerve Impairment
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TRIAD
Total posts in Category: 25
Interim Analysis Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)
Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)
SORD: A Lesson in Repurposing Drugs to Treat Hereditary Neuropathy
Research from a mutation on the SORD gene
TRPV4 Hereditary Neuropathy Patients Connect with Johns Hopkins Experts to Push Research Efforts Forward
Community connected with the TRPV4 HN team at Johns Hopkins
CMT1A Clinical Trial Update: Pharnext PREMIER trial of PXT3003
The PREMIER Trial, which is being conducted in patients with mild-to-moderate CMT1A, is expected to enroll approximately 350 subjects ages 16-65 with a confirmed genetic diagnosis of CMT1A.
Mouse Models of Human CNTNAP1 Mutations: Strategies to Rescue and Restore Functions
Research with the Department of Cellular and Integrative Physiology UT Health San Antonio, TX
Free CMT Genetic Testing for a Common Axonal type of CMT to Prepare for Clinical Trial
A new type of Charcot-Marie-Tooth (CMT) has recently been identified caused by mutations in the SORD gene.
3 Reasons to Join the Global Registry for Inherited Neuropathies – GRIN!
What GRIN is and why you should join
CMT&Me COMPETITION
Participating during CMT month
US Food and Drug Administration has agreed with Pharnext and provided clear guidance on the regulatory pathway to approval for PXT3003, including key design elements of a single pivotal Phase III study
The FDA has agreed with the key elements of Pharnext’s approach for the developmental pathway to approval for PXT3003.
The Miracle We’ve Been Praying For!
Alana Kohler’s story of having CMT4A.
Press Release: Pharnext provides regulatory and clinical update on PXT3003 Phase III study for the treatment of Charcot-Marie-Tooth Type 1A
US Food and Drug Administration has agreed with Pharnext and provided clear guidance on the regulatory pathway to approval for PXT3003, including key design elements of a single pivotal Phase III study
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