The Niemann-Pick Disease Type C drugs in development market research report provides comprehensive information on the therapeutics under development for Niemann-Pick Disease Type C, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. GlobalData’s report assesses key aspects of the companies and drugs in development for Niemann-Pick Disease Type C. Buy the report here.
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The report also covers the descriptive pharmacological action of the therapeutics and the latest news and press releases. Additionally, the report provides an overview of the key players involved in therapeutic development for Niemann-Pick Disease Type C and features dormant and discontinued products.
GlobalData tracks 32 drugs in development for Niemann-Pick Disease Type C by 30 companies/universities/institutes. The top development phase for Niemann-Pick Disease Type C is preclinical with 18 drugs in that stage. The Niemann-Pick Disease Type C pipeline has 27 drugs in development by companies and five by universities/ institutes. Some of the companies in the Niemann-Pick Disease Type C pipeline products market are: IntraBio, StrideBio and Scenic Biotech.
The key targets in the Niemann-Pick Disease Type C pipeline products market include Niemann Pick C1 Protein (NPC1), Ceramide Glucosyltransferase (Glucosylceramide Synthase or GLCT 1 or UDP Glucose:N Acylsphingosine D Glucosyltransferase or UDP Glucose Ceramide Glucosyltransferase or UGCG or EC 2.4.1.80), and Cholesterol.
The key mechanisms of action in the Niemann-Pick Disease Type C pipeline product include Niemann Pick C1 Protein (NPC1) Activator with five drugs in Preclinical. The Niemann-Pick Disease Type C pipeline products include six routes of administration with the top ROA being Intravenous and seven key molecule types in the Niemann-Pick Disease Type C pipeline products market including Small Molecule, and Gene Therapy.
Niemann-Pick Disease Type C overview
Niemann-Pick disease is one of a group of lysosomal storage diseases that affect metabolism and that are caused by genetic mutations. It involves the accumulation of sphingolipids in cells throughout the body, particularly reticuloendothelial cells (the mononuclear phagocyte system). Symptoms include enlarged liver, brain damage, difficulty walking and swallowing, increased sensitivity to touch, difficulty speaking, loss of muscle tone, and learning difficulties.
For a complete picture of Niemann-Pick Disease Type C’s pipeline drug market, buy the report here.
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GlobalData’s pipeline drugs offers detailed profiles of pharmaceutical drugs in all stages of pre-clinical and clinical development, from discovery through to pre-registration. Coverage is limited to novel human medicinal drugs and biosimilars seeking market approval proprietary and is one of two primary repositories of pharmaceutical drug information offered by GlobalData through its Pharmaceutical Intelligence Center.
