The Methylmalonic Acidemia drugs in development market research report provides comprehensive information on the therapeutics under development for Methylmalonic 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 Methylmalonic Acidemia. 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 Methylmalonic Acidemia and features dormant and discontinued products.
GlobalData tracks ten drugs in development for Methylmalonic Acidemia by eight companies/universities/institutes. The top development phase for Methylmalonic Acidemia is preclinical with five drugs in that stage. The Methylmalonic Acidemia pipeline has ten drugs in development by companies and 0 by universities/ institutes. Some of the companies in the Methylmalonic Acidemia pipeline products market are: HemoShear Therapeutics, Agios Pharmaceuticals and Logicbio Therapeutics.
The key targets in the Methylmalonic Acidemia pipeline products market include Methylmalonyl CoA Mutase Mitochondrial (Methylmalonyl CoA Isomerase or MCM or MUT or EC 5.4.99.2), Pantothenate Kinase 2 Mitochondrial (Pantothenic Acid Kinase 2 or PANK2 or EC 2.7.1.33), and Branched Chain Amino Acid Aminotransferase Mitochondrial (Placental Protein 18 or BCAT2 or EC 2.6.1.42).
The key mechanisms of action in the Methylmalonic Acidemia pipeline product include Methylmalonyl CoA Mutase Mitochondrial (Methylmalonyl CoA Isomerase or MCM or MUT or EC 5.4.99.2) Activator with two drugs in Phase II. The Methylmalonic Acidemia pipeline products include two routes of administration with the top ROA being Oral and three key molecule types in the Methylmalonic Acidemia pipeline products market including Small Molecule, and Gene Therapy.
Methylmalonic Acidemia overview
Methylmalonic Acidemia is an inborn or inherited metabolic disease where the levels of MMA (methylmalonic acid) are elevated in blood and urine. Deficiency in MMA Defect in conversion of methylmalonyl-coenzyme A (CoA) to succinyl-CoA causes accumulation of methylmalonyl-CoA and other potentially toxic compounds. It can be mild to severe appearing in early stages of life or in later stages. Seizures, encephalopathy and stroke are the neurological complication associated and if left untreated leads to coma and death. Vomiting’s, dehydration, lethargy, are other symptoms. Molecular genetic testing is a diagnostic method to find out the mutations on the gene. Protein restricted diet, high calorie diet followed by vitamin B12 supplementation, antibiotics and organ transplantation are other strategies.
For a complete picture of Methylmalonic Acidemia’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.
