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 11 drugs in development for Propionic Acidemia by eight companies/universities/institutes. The top development phase for Propionic Acidemia is preclinical with seven drugs in that stage. The Propionic Acidemia pipeline has nine drugs in development by companies and two by universities/ institutes. Some of the companies in the Propionic Acidemia pipeline products market are: HemoShear Therapeutics, Imbria Pharmaceuticals and National Institutes of Health.
The key targets in the Propionic Acidemia pipeline products market include Propionyl CoA Carboxylase Alpha Chain Mitochondrial (Propanoyl CoA Carbon Dioxide Ligase Subunit Alpha or PCCA or EC 6.4.1.3), Propionyl CoA Carboxylase Beta Chain Mitochondrial (Propanoyl CoA Carbon Dioxide Ligase Subunit Beta or PCCB or EC 6.4.1.3), and Pantothenate Kinase 2 Mitochondrial (Pantothenic Acid Kinase 2 or PANK2 or EC 2.7.1.33).
The key mechanisms of action in the Propionic Acidemia pipeline product include Propionyl CoA Carboxylase Alpha Chain Mitochondrial (Propanoyl CoA Carbon Dioxide Ligase Subunit Alpha or PCCA or EC 6.4.1.3) Activator with three drugs in Phase II. The Propionic Acidemia pipeline products include two routes of administration with the top ROA being Oral and two key molecule types in the Propionic Acidemia pipeline products market including Small Molecule, and Gene Therapy.
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.
