The GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency) drugs in development market research report provides comprehensive information on the therapeutics under development for GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency), 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 GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency). 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 GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency) and features dormant and discontinued products.
GlobalData tracks 15 drugs in development for GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency) by 12 companies/universities/institutes. The top development phase for GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency) is preclinical with nine drugs in that stage. The GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency) pipeline has 15 drugs in development by companies and 0 by universities/ institutes. Some of the companies in the GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency) pipeline products market are: Gain Therapeutics, Liberyx Therapeutics and Sanofi.
The key targets in the GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency) pipeline products market include Beta Galactosidase (Acid Beta Galactosidase or Lactase or Elastin Receptor 1 or GLB1 or EC 3.2.1.23), and Ceramide Glucosyltransferase (Glucosylceramide Synthase or GLCT 1 or UDP Glucose:N Acylsphingosine D Glucosyltransferase or UDP Glucose Ceramide Glucosyltransferase or UGCG or EC 2.4.1.80).
The key mechanisms of action in the GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency) pipeline product include Beta Galactosidase (Acid Beta Galactosidase or Lactase or Elastin Receptor 1 or GLB1 or EC 3.2.1.23) Activator with five drugs in Phase II. The GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency) pipeline products include three routes of administration with the top ROA being Oral and four key molecule types in the GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency) pipeline products market including Small Molecule, and Enzyme.
GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency) overview
GM1 gangliosidosis is a rare lysosomal storage disorder characterized biochemically by deficient beta-galactosidase activity and clinically by a wide range of variable neurovisceral, ophthalmological and dysmorphic features. The disorder is caused by mutations in the GLB1gene (3p22.3) coding for beta-galactosidase. To date, more than 165 mutations have been identified. Deficient enzyme activity leads to toxic accumulation of gangliosides in body tissues, and particularly in the central nervous system (CNS).
For a complete picture of GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency)’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.
