Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Drugs In Development, 2024
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Acid sphingomyelinase deficiency (ASMD) is a rare genetic disorder that affects the metabolism of a lipid called sphingomyelin. ASMD is caused by mutations in the SMPD1 gene, which encodes the enzyme acid sphingomyelinase (ASM). ASM is responsible for breaking down sphingomyelin in the lysosomes of cells. When ASM is deficient, sphingomyelin accumulates in various tissues and organs, causing damage and dysfunction. ASMD is also known as Niemann-Pick disease types A and B, depending on the severity and presence of neurological symptoms. ASMD can cause hepatosplenomegaly, thrombocytopenia, ataxia, dystonia, dementia, and bone abnormalities. ASMD is inherited in an autosomal recessive manner and has no cure. Treatment is supportive and symptomatic.
The Niemann-Pick Disease drugs in development market research report provide comprehensive information on the therapeutics under development for Niemann-Pick Disease, 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 and features dormant and discontinued products.
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Scope
- Therapeutics in Development: Covering 40 molecules, with 33 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 therapeutics, aiding strategic decision-making with insights into stages, targets, MoA, RoA and molecule types.
- Pipeline Exploration: Explore the detailed Niemann-Pick Disease 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 treatments for adaptable decision-making.
- Save valuable hours: Identify key players steering innovation in therapeutics, enabling strategic partnerships.
Key Players
Azafaros BVBe Biopharma Inc
Bloomsbury Genetic Therapies Ltd
Chameleon Biosciences Inc
Cyclo Therapeutics Inc
CycloLab Cyclodextrin Research and Development Laboratory Ltd
Deep Genomics Inc
Duke University
Edenbridge Pharmaceuticals LLC
Endece LLC
Evox Therapeutics Ltd
Gene Therapy Research Institution Co Ltd
IntraBio Ltd
Kumamoto University
Mandos LLC
Merck & Co Inc
NeuImmune Inc
Okklo Life Sciences BV
Oragenics Inc
Oraxion Therapeutics Inc
Oregon State University
Orphazyme A/S
Perlara PBC
Polaryx Therapeutics Inc
Prevacus Inc
ProfoundBio Suzhou Co Ltd
Sanofi
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
University of California San Francisco
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