Charcot-Marie-Tooth Disease Pipeline
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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Sep 03, 2021
Charcot-Marie-Tooth Disease Market Size Expects Growth Owing to Increasing Prevalence, Influx of Pharma Companies and Novel Therapies
Charcot-Marie-Tooth (CMT) disease is a rare, chronic and sensory neuropathy that causes weakness of the limbs and progresses gradually over time. It can lead to atrophy, sensory loss and complete limb dysfunction. However, rare, the condition is the most common inherited disorder that damages peripheral nerves with...
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