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Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development in DENMARK


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            Lead Product(s): Arimoclomol

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 28, 2021


            NPC patients receiving routine clinical care was estimated to be a mean increase of 5.2 points after three years, based on a statistical model combining placebo data from the NPC-002 double-blind study and prospective data from the observational NPC-001 study.