Charcot-Marie-Tooth Disease Pipeline
Jul 10, 2026
The Race to Transform Charcot-Marie-Tooth Disease Treatment: Which Therapy Will Cross the Finish Line First?
Summary Charcot-Marie-Tooth disease is genetically heterogeneous, with more than 100 genes associated with different CMT disease subtypes. Currently, no approved therapies are available for the treatment of CMT disease, leaving patients with limited treatment options. As a result, there remains a significant...
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Mar 18, 2025
FDA Expands SOLIRIS for Pediatric Myasthenia Gravis; vTv’s Cadisegliatin Program Resumes as FDA Lifts Hold; ENCell’s EN001 Wins Orphan Drug Designation for Charcot-Marie-Tooth; FDA Accepts Sydnexis’ NDA for SYD-101 in Pediatric Myopia; Cambium Bio’s Potency Assay Strategy Cleared for Elate Ocular Phase III Trials
MDA Applauds FDA’s Expanded Approval of Soliris for Pediatric Generalized Myasthenia Gravis The Muscular Dystrophy Association (MDA) celebrates the FDA approval of an expanded indication for Alexion/AstraZeneca’s eculizumab (Soliris), now authorized for pediatric patients aged six and older with generalized myas...
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