Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Drugs In Development, 2024
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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.
The Niemann-Pick Disease Type C drugs in development market research report provide 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. The report also covers the descriptive pharmacological action and product description of the therapeutics, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Niemann-Pick Disease Type C and features dormant and discontinued products.
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* Certain sections in the report may be removed or altered based on the availability and relevance of data.
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Scope
- Therapeutics in Development: Covering 32 molecules, with 27 developed by companies and the rest by universities/institutes.
- Analysis Parameters: Offering insights by stage of development, drug target, MoA, RoA, and molecule type for a comprehensive overview.
- Pharmacological Insights: Understand the descriptive pharmacological action of therapeutics.
- Development History: Access the complete research and development history.
- Latest News and Press Releases: Stay updated with the latest developments through comprehensive news coverage.
Reasons to Buy
- Holistic insights: Understand the broad spectrum of Niemann-Pick Disease Type C therapeutics, aiding strategic decision-making with insights into stages, targets, MoA, RoA and molecule types.
- Pipeline Exploration: Explore the detailed Niemann-Pick Disease Type C pipeline, offering nuanced analysis of drug targets, mechanisms of action and routes of administration for varied decision-making.
- Comprehensive R&D: Access thorough R&D histories, providing a comprehensive understanding of Niemann-Pick Disease Type C treatments for adaptable decision-making.
- Save valuable hours: Identify key players steering innovation in therapeutics, enabling strategic partnerships.
Key Players
Azafaros BVBloomsbury Genetic Therapies Ltd
Chameleon Biosciences Inc
Cyclo Therapeutics Inc
CycloLab Cyclodextrin Research and Development Laboratory Ltd
Deep Genomics Inc
Edenbridge Pharmaceuticals LLC
Endece LLC
Evox Therapeutics Ltd
Gene Therapy Research Institution Co Ltd
IntraBio Ltd
Kumamoto University
Mandos LLC
Merck & Co Inc
Okklo Life Sciences BV
Oragenics Inc
Oraxion Therapeutics Inc
Oregon State University
Orphazyme A/S
Perlara PBC
Prevacus Inc
ProfoundBio Suzhou Co Ltd
Scenic Biotech BV
Shanghai Pharmaceuticals Holding Co Ltd
SOM Biotech SL
StrideBio Inc
Synaptogenix Inc
Tokyo Medical and Dental University
U.S. National Institutes of Health
University Hospitals Leuven
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