The Propionic Acidemia drugs in development market research report provides comprehensive information on the therapeutics under development for Propionic Acidemia, 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 Propionic Acidemia. 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 Propionic Acidemia and features dormant and discontinued products.
GlobalData tracks five drugs in development for Propionic Acidemia by five companies/universities/institutes. The top development phase for Propionic Acidemia is preclinical with three drugs in that stage. The Propionic Acidemia pipeline has two drugs in development by companies and three by universities/ institutes. Some of the companies in the Propionic Acidemia pipeline products market are: Ironwood Pharmaceuticals, University of Pittsburgh and Moderna.
The key targets in the Propionic Acidemia pipeline products market include Propionyl CoA Carboxylase Alpha Chain Mitochondrial, and Propionyl CoA Carboxylase Beta Chain Mitochondrial.
The key mechanisms of action in the Propionic Acidemia pipeline product include Propionyl CoA Carboxylase Alpha Chain Mitochondrial Activator with three drugs in Phase II. The Propionic Acidemia pipeline products include one routes of administration with the top ROA being Intravenous and two key molecule types in the Propionic Acidemia pipeline products market including Gene Therapy, and Small Molecule.
Propionic Acidemia overview
The spectrum of propionic acidemia (PA) ranges from neonatal-onset to late-onset disease. Neonatal-onset PA is characterized by a healthy newborn with poor feeding and decreased arousal in the first few days of life, followed by progressive encephalopathy of unexplained origin. Without prompt diagnosis, this is followed by progressive encephalopathy manifesting as lethargy, seizures, or coma that can result in death. Individuals with late-onset PA may remain asymptomatic and suffer a metabolic crisis under catabolic stress (e.g., illness, surgery, fasting) or may experience a more insidious onset with the development of multiorgan complications including vomiting, protein intolerance, failure to thrive, hypotonia, developmental delays or regression, movement disorders, or cardiomyopathy.
For a complete picture of Propionic Acidemia’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.
