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.

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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 Maple Syrup Urine Disease and features dormant and discontinued products.

GlobalData tracks 11 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 five drugs in that stage. The Maple Syrup Urine Disease pipeline has 11 drugs in development by companies and 0 by universities/ institutes. Some of the companies in the Maple Syrup Urine Disease pipeline products market are: Syntis Bio, Darling Therapeutics and Moderna.

The key targets in the Maple Syrup Urine Disease pipeline products market include 5-Hydroxytryptamine Receptor 1A, [3 Methyl 2 Oxobutanoate Dehydrogenase [Lipoamide]] Kinase Mitochondrial, and Enteropeptidase.

The key mechanisms of action in the Maple Syrup Urine Disease pipeline product include 5-Hydroxytryptamine Receptor 1A 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 six key molecule types in the Maple Syrup Urine Disease pipeline products market including Small Molecule, and Gene Therapy.

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.

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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.