The Lipid Storage Disorders (Lipidoses) drugs in development market research report provides comprehensive information on the therapeutics under development for Lipid Storage Disorders (Lipidoses), 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 Lipid Storage Disorders (Lipidoses). 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 Lipid Storage Disorders (Lipidoses) and features dormant and discontinued products.
GlobalData tracks 138 drugs in development for Lipid Storage Disorders (Lipidoses) by 102 companies/universities/institutes. The top development phase for Lipid Storage Disorders (Lipidoses) is preclinical with 69 drugs in that stage. The Lipid Storage Disorders (Lipidoses) pipeline has 123 drugs in development by companies and 15 by universities/ institutes. Some of the companies in the Lipid Storage Disorders (Lipidoses) pipeline products market are: Gain Therapeutics, Takeda Pharmaceutical and M6P Therapeutics.
The key targets in the Lipid Storage Disorders (Lipidoses) pipeline products market include Glucosylceramidase (Acid Beta Glucosidase or Alglucerase or D Glucosyl N Acylsphingosine Glucohydrolase or Beta Glucocerebrosidase or Imiglucerase or GBA or EC 3.2.1.45), Alpha Galactosidase A (Alpha D-Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22), 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 Lipid Storage Disorders (Lipidoses) pipeline product include Alpha Galactosidase A (Alpha D Galactosidase A or Alpha D Galactoside Galactohydrolase or Melibiase or Agalsidase or GLA or EC 3.2.1.22) Replacement with 14 drugs in Phase III. The Lipid Storage Disorders (Lipidoses) pipeline products include ten routes of administration with the top ROA being Intravenous and 16 key molecule types in the Lipid Storage Disorders (Lipidoses) pipeline products market including Small Molecule, and Gene Therapy.
Lipid Storage Disorders (Lipidoses) overview
Lipid storage disorders, or lipidoses, encompass inherited metabolic conditions where fats accumulate due to enzyme deficiencies, impacting various organs. Gaucher, Niemann-Pick, Tay-Sachs, Fabry, and Krabbe diseases are examples. Symptoms, varying by disorder, may include neurological problems, organ enlargement, and skeletal abnormalities. Treatments focus on symptom management, employing enzyme replacement, substrate reduction therapies, and supportive care. Managing these complex conditions requires tailored approaches; however, some lack specific treatments. Genetic counseling aids in understanding inheritance patterns and risks for affected families, offering guidance amidst these rare and diverse disorders.
For a complete picture of Lipid Storage Disorders (Lipidoses)’s pipeline drug market, buy the report here.
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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.
