The Glycogen Storage Disease 1A drugs in development market research report provides comprehensive information on the therapeutics under development for Glycogen Storage Disease 1A, 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 Glycogen Storage Disease 1A. 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 Glycogen Storage Disease 1A and features dormant and discontinued products.
GlobalData tracks eight drugs in development for Glycogen Storage Disease 1A by seven companies/universities/institutes. The top development phase for Glycogen Storage Disease 1A is preclinical with seven drugs in that stage. The Glycogen Storage Disease 1A pipeline has eight drugs in development by companies and 0 by universities/ institutes. Some of the companies in the Glycogen Storage Disease 1A pipeline products market are: Beam Therapeutics, Liberyx Therapeutics and Ultragenyx Pharmaceutical.
The key targets in the Glycogen Storage Disease 1A pipeline products market include Glucose 6 Phosphatase (G6Pase or Glucose 6 Phosphatase Alpha or G6PC or EC 3.1.3.9), ATP Sensitive Inward Rectifier Potassium Channel 11 (Potassium Channel Inwardly Rectifying Subfamily J Member 11 or Inward Rectifier Potassium Channel Kir6.2 or KCNJ11), and Peroxisome Proliferator Activated Receptor Alpha (Nuclear Receptor Subfamily 1 Group C Member 1 or NR1C1 or PPARA).
The key mechanisms of action in the Glycogen Storage Disease 1A pipeline product include Glucose 6 Phosphatase (G6Pase or Glucose 6 Phosphatase Alpha or G6PC or EC 3.1.3.9) Activator with six drugs in Phase III. The Glycogen Storage Disease 1A pipeline products include three routes of administration with the top ROA being Intravenous and four key molecule types in the Glycogen Storage Disease 1A pipeline products market including Gene Therapy, and Small Molecule.
Glycogen Storage Disease 1A overview
Glycogen storage disease type I (GSD I), also known as Von Gierke disease, is an inherited disorder caused by deficiencies of specific enzymes in the glycogen metabolism pathway. It comprises two major subtypes, GSD Ia and GSD Ib. In GSD Ia, there is a deficiency of enzyme glucose-6-phosphatase (G6Pase), which cleaves glycogen to glucose, thus leading to hypoglycemia and lactic acidosis.
For a complete picture of Glycogen Storage Disease 1A’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.
