The Angelman Syndrome drugs in development market research report provides 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. GlobalData’s report assesses key aspects of the companies and drugs in development for Angelman Syndrome. Buy the report here.
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The report also covers the descriptive pharmacological action of the therapeutics and the latest news and press releases. Additionally, the report provides an overview of the key players involved in therapeutic development for Angelman Syndrome and features dormant and discontinued products.
GlobalData tracks 23 drugs in development for Angelman Syndrome by 23 companies/universities/institutes. The top development phase for Angelman Syndrome is preclinical with eight drugs in that stage. The Angelman Syndrome pipeline has 23 drugs in development by companies and 0 by universities/ institutes. Some of the companies in the Angelman Syndrome pipeline products market are: Neuren Pharmaceuticals, CuroNZ and Astellas Pharma.
The key targets in the Angelman Syndrome pipeline products market include Ubiquitin Protein Ligase E3A, Transient Receptor Potential Cation Channel Subfamily V Member 1, and Sigma Non Opioid Intracellular Receptor 1.
The key mechanisms of action in the Angelman Syndrome pipeline product include Ubiquitin Protein Ligase E3A Activator with eight drugs in Preclinical. The Angelman Syndrome pipeline products include six routes of administration with the top ROA being Oral and seven key molecule types in the Angelman Syndrome pipeline products market including Gene Therapy, and Small Molecule.
Angelman Syndrome overview
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.
For a complete picture of Angelman Syndrome’s pipeline drug market, buy the report here.
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GlobalData’s pipeline drugs offers detailed profiles of pharmaceutical drugs in all stages of pre-clinical and clinical development, from discovery through to pre-registration. Coverage is limited to novel human medicinal drugs and biosimilars seeking market approval proprietary and is one of two primary repositories of pharmaceutical drug information offered by GlobalData through its Pharmaceutical Intelligence Center.
