The Maple Syrup Urine Disease drugs in development market research report provides comprehensive information on the therapeutics under development for Maple Syrup Urine Disease, 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 Maple Syrup Urine Disease. Buy the report here.
Smarter leaders trust GlobalData
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 Maple Syrup Urine Disease and features dormant and discontinued products.
GlobalData tracks ten drugs in development for Maple Syrup Urine Disease by ten companies/universities/institutes. The top development phase for Maple Syrup Urine Disease is preclinical with six drugs in that stage. The Maple Syrup Urine Disease pipeline has ten drugs in development by companies and 0 by universities/ institutes. Some of the companies in the Maple Syrup Urine Disease pipeline products market are: Relief Therapeutics, LyGenesis and ASC Therapeutics.
The key targets in the Maple Syrup Urine Disease pipeline products market include Glutamine, 5-Hydroxytryptamine Receptor 1A (5 HT1A or G 21 or Serotonin Receptor 1A or HTR1A), and [3 Methyl 2 Oxobutanoate Dehydrogenase [Lipoamide]] Kinase Mitochondrial (Branched Chain Alpha Ketoacid Dehydrogenase Kinase or BCKDHKIN or BCKDK or EC 2.7.11.4).
The key mechanisms of action in the Maple Syrup Urine Disease pipeline product include 5-Hydroxytryptamine Receptor 1A (5 HT1A or G 21 or Serotonin Receptor 1A or HTR1A) Antagonist with one drug in Phase I. The Maple Syrup Urine Disease pipeline products include three routes of administration with the top ROA being Oral and five key molecule types in the Maple Syrup Urine Disease pipeline products market including Gene Therapy, and Small Molecule.
Maple Syrup Urine Disease overview
Maple Syrup Urine Disease is a rare inherited metabolic disease caused by a deficiency of an enzyme alpha-ketoacid dehydrogenase complex (BCKD) as a result accumulation of proteins harmful proteins or certain amino acids in blood and urine are found. Predominant in infants and older groups. Sweet-smelling urine, loss of appetite, anorexia, and breathing difficulties are common symptoms. If diagnosed in infant emergency treatment is initiated to prevent complications. Whole blood picture, genetic mutations on BCKDHA, BCKDHB. Dietary leucine restriction, liver transplantation, and non-pharmacological methods are low protein diet, preventive measures, and genetic counseling.
For a complete picture of Maple Syrup Urine Disease’s pipeline drug market, buy the report here.
Data Insights
From
The gold standard of business intelligence.
Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.
GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.
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.
