Niemann Pick Type-C Disease Pipeline Overview
Niemann Pick Type-C Disease (NPC) is a progressive autosomal recessive genetic disorder of lysosome storage due to an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells. The accumulation of these substances damages the affected areas. It can range from a fatal disorder within the first few months after birth (neonatal period) to a late onset, chronic progressive disorder that remains undiagnosed well into adulthood. It affects both males and females equally. NPC is estimated to occur in 1 in 100,000-120,000 live births.
The onset of symptoms may begin at any age, most commonly in school age children; however, infants, adolescents and adults can all be affected by the disease. With the progression of disease, children may experience developmental delays, challenges with motor skills (e.g. increased clumsiness and abnormal posturing) and difficulty in making vertical eye movements. Dystonia and seizures are common in the patients suffering from NPC.
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Several tests are available for the diagnosis of NPC. These tests include biochemical testing, molecular genetic testing, NPC screening using biomarkers among others. No marketed drugs are currently present for NPC in the U.S. The drug development companies are involved in quest to develop an effective drug and better treatment strategies. There are several companies utilizing their proprietary technologies for the development of effective and better drugs.
The therapeutic pipeline of NPC Disease consists of approximately 9+ products in different stages of development. Currently 2+ drugs are in Phase III development and major drugs are in pre-clinical and discovery stage.
Top Company Analysed
Some of the key players include Orphazyme, CTD Holdings, Mallinckrodt Pharmaceuticals, IntraBio, Perlara, Neurotrope BioScience, Evox Therapeutics, Okklo Life Sciences, Amicus Therapeutics.
Scope of the report:
• Provides an overview of therapeutic pipeline activity for Niemann Pick Type-C Disease across the complete product development cycle including all clinical and non-clinical stages
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• The report comprises of detailed description of Niemann Pick Type-C Disease therapeutic products with key coverage of developmental activities including licensing & collaboration deals, patents issued, designations, technologies and chemical information
• Therapeutic assessment of the active pipeline products by stage, product type, molecule type, and route of administration
Reasons to buy:
• The Niemann Pick Type-C Disease (NPC) pipeline presents the detailed profile of drugs. The analysis offered in the report is a combination of deep dive secondary research and input from Key Opinion Leader of the industry
• The report presents a quick review of the current scenario regarding the drug development of the indication at one glance
• The report covers in-depth analysis of prominent industry peers with a primary focus on company consolidation, designation, technology, agreements and patents regarding the therapy
• Detailed examination on diagnosis, treatment and guidelines prevailing in the industry
• Examination of industry attractiveness with the help of approval timelines
• The study comprehensively covers the market across drugs in different phases of development
The Niemann Pick Type-C Disease pipeline analysis report can be customized to the country level or any other competitive segment. Besides this, UMI understands that you may have your own business need, hence we also provide fully customized solutions to clients.
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