Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type A – Drugs In Development, 2024
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Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disease and an autosomal recessive genetic disorder characterized by the deficiency of sphingomyelinase. It is caused by a mutation of the SMPD1 gene. The subtypes of ASMD include Niemann-Pick disease (NPD) types A and B. Due to the deficiency of sphingomyelinase, sphingomyelin accumulates in the brain, kidneys, liver, and other organs. Enlargement of the liver and spleen, feeding difficulties, loss of reflexes, and cherry red spot macula in infants can be seen in patients with ASMD. It can be diagnosed through molecular genetic testing of the SMPD1 gene. Olipudase alfa (Xenpozyme) enzyme replacement therapy (ERT) helps to reduce the accumulation of sphingomyelin.
The Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) drugs in development market research report provide comprehensive information on the therapeutics under development for Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency), 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 A (Acid Sphingomyelinase Deficiency) and features dormant and discontinued products.
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Scope
- Therapeutics in Development: Covering 7 molecules, with 5 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 A (Acid Sphingomyelinase Deficiency) therapeutics, aiding strategic decision-making with insights into stages, targets, MoA, RoA and molecule types.
- Pipeline Exploration: Explore the detailed Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) 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 A (Acid Sphingomyelinase Deficiency) treatments for adaptable decision-making.
- Save valuable hours: Identify key players steering innovation in therapeutics, enabling strategic partnerships.
Key Players
Duke UniversityIntraBio Ltd
Polaryx Therapeutics Inc
University of California San Francisco
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