Angelman Syndrome – Drugs In Development, 2024
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Angelman syndrome, an intricate genetic disorder primarily impacting the nervous system, manifests with distinctive features such as delayed development, intellectual disability, severe speech impairment, and ataxia, involving movement and balance issues. Recurrent seizures (epilepsy) and microcephaly, characterized by a small head size, are also prevalent in affected children. Recognizable at 6 to 12 months of age, delayed development intensifies in early childhood. With an incidence of 1 in 12,000 to 20,000 individuals, Angelman syndrome’s key attributes stem from the functional loss of the UBE3A gene. Normally, individuals inherit an active copy of UBE3A from both parents, but in nerve cells, only the maternal copy functions due to genomic imprinting. Loss of the maternal copy due to chromosomal changes or mutations leaves most brain areas without an active UBE3A gene.
The Angelman Syndrome drugs in development market research report provide comprehensive information on the therapeutics under development for Angelman Syndrome, 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 Angelman Syndrome and features dormant and discontinued products.
Note:
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Scope
- Therapeutics in Development: Covering 22 molecules, with 22 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 Angelman Syndrome therapeutics, aiding strategic decision-making with insights into stages, targets, MoA, RoA and molecule types.
- Pipeline Exploration: Explore the detailed Angelman Syndrome 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 Angelman Syndrome treatments for adaptable decision-making.
- Save valuable hours: Identify key players steering innovation in therapeutics, enabling strategic partnerships.
Key Players
Amicus Therapeutics IncAnavex Life Sciences Corp
Angelini Pharma Inc
Astellas Pharma Inc
Biom Pharmaceutical Corp
Biom Therapeutics LLC
Couragene Inc
CuroNZ Ltd
Denali Therapeutics Inc
Exicure Inc
F. Hoffmann-La Roche Ltd
GEXVal Inc
Healx Ltd
Ionis Pharmaceuticals Inc
Modalis Therapeutics Corp
Neuren Pharmaceuticals Ltd
Ovid Therapeutics Inc
Radius Health Inc
StrideBio Inc
Taysha Gene Therapies Inc
Ultragenyx Pharmaceutical Inc
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