New Drug Application for Niemann-Pick Disease Type C Treatment Submitted Under Rolling Review
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New Drug Application for Niemann-Pick Disease Type C Treatment Submitted Under Rolling Review

According to a story from globenewswire.com, the biopharmaceutical company Orphazyme A/S has recently announced that it has submitted its New Drug Application (NDA) for its experimental treatment arimoclomol to the…

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Family Affected by Niemann-Pick Disease Type C Faces Challenges During COVID-19
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Family Affected by Niemann-Pick Disease Type C Faces Challenges During COVID-19

According to a story from Spectrum News, the Andrews family has been dealing with a rare disease called Niemann-Pick disease type C for years. Parents Pam and Chris knew early…

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Study: Arimoclomol Shows Positive Results in the Treatment of Niemann-Pick Disease
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Study: Arimoclomol Shows Positive Results in the Treatment of Niemann-Pick Disease

  Orphazyme, a biopharmaceutical company, has recently released the data from their study of arimoclomol. This product is meant for the treatment of Niemann-Pick disease type C (NPC). Orphazyme specializes…

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This Company Just Provided a Grant for a Niemann-Pick Disease Type C Screening Program
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This Company Just Provided a Grant for a Niemann-Pick Disease Type C Screening Program

According to a story from ir.stockpr.com, the biotechnology company Cyclo Therapeutics, Inc. (previously known as CTD Holdings) has recently thrown its support behind a Niemann-Pick Disease Type C newborn screening…

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ICYMI: FDA Approves Expanded Access Application of Experimental Niemann-Pick Type C Drug
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ICYMI: FDA Approves Expanded Access Application of Experimental Niemann-Pick Type C Drug

According to a press release from CTD Holdings, the American Food and Drug Administration (FDA) recently approved an Expanded Access application from a physician to treat a pediatric Niemann-Pick disease…

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FDA Approves a Program for Expanded Treatment Access in Niemann-Pick Disease Type C
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FDA Approves a Program for Expanded Treatment Access in Niemann-Pick Disease Type C

According to a story from ir.stockpr.com, the biotechnology company CTD Holdings, Inc., recently announced that the US Food and Drug Administration (FDA) has issued an approval for an individual Investigational…

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CTD Holdings Gives Presentation on Investigatory Niemann-Pick Type C Drug at Family Support and Medical Conference

According to a press release from CTD Holdings, a biotechnology company based in Gainesville, Florida, the Company recently presented data collected from their investigatory Niemann-Pick Disease treatment, Trappsol Cyclo, to…

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