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Des infos fraiches
Significant portion of core target patient population already located
- ~26,000 CMT1A patients located in the US and EU5 through market research, claims data analysis, field
activities and PAGs
- Several patient registries available: GRIN, INC, CMT-Net, etc.
- Plans to go beyond prior launch with MSLs and sponsored Dx campaign
▪ Primary target treating physicians identified (cover >80% of CMT patients)
- Neurologists (NMD specialists), GPs, podiatrists, PTs
▪ Strong relationships developed with key stakeholders
▪ Patient advocacy groups
▪ KOLs and scientific societies
▪ Generation of pharmacoeconomic data prior to launch
▪ CMT&Me: digital lifestyle study sponsored by Pharnext (>2,600 patients enrolled)
Continual assessment of commercial partnership options for various geographies, including US, EU,
Japan and ROW; China commercialization rights are licensed to GeneNet, a JV formed by Pharnext & Tasly
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