The Sandhoff Disease (Jatzkewitz-Pilz Syndrome) drugs in development market research report provides comprehensive information on the therapeutics under development for Sandhoff Disease (Jatzkewitz-Pilz Syndrome), 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 Sandhoff Disease (Jatzkewitz-Pilz Syndrome). Buy the report here.

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 Sandhoff Disease (Jatzkewitz-Pilz Syndrome) and features dormant and discontinued products.

GlobalData tracks 13 drugs in development for Sandhoff Disease (Jatzkewitz-Pilz Syndrome) by 11 companies/universities/institutes. The top development phase for Sandhoff Disease (Jatzkewitz-Pilz Syndrome) is preclinical with eight drugs in that stage. The Sandhoff Disease (Jatzkewitz-Pilz Syndrome) pipeline has ten drugs in development by companies and three by universities/ institutes. Some of the companies in the Sandhoff Disease (Jatzkewitz-Pilz Syndrome) pipeline products market are: Orphi Therapeutics, Polaryx Therapeutics and Azafaros.

The key targets in the Sandhoff Disease (Jatzkewitz-Pilz Syndrome) pipeline products market include 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), Beta-Hexosaminidase Subunit Alpha (Beta-N-Acetylhexosaminidase Subunit Alpha or N-Acetyl-Beta-Glucosaminidase Subunit Alpha or HEXA or EC 3.2.1.52), and Protein O GlcNAcase (Beta N Acetylglucosaminidase or Beta N Acetylhexosaminidase or Beta Hexosaminidase or OGA or EC 3.2.1.169).

The key mechanisms of action in the Sandhoff Disease (Jatzkewitz-Pilz Syndrome) pipeline product include 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) Inhibitor with three drugs in Phase III. The Sandhoff Disease (Jatzkewitz-Pilz Syndrome) pipeline products include three routes of administration with the top ROA being Oral and four key molecule types in the Sandhoff Disease (Jatzkewitz-Pilz Syndrome) pipeline products market including Small Molecule, and Gene Therapy.

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) overview

Sandhoff disease is an inherited lipid storage disorder that progressively destroys nerve cells (neurons) in the brain and spinal cord. It is caused by a deficiency of the enzyme beta-hexosaminidase. Symptoms include progressive nervous system deterioration, problems initiating and controlling muscles and movement, increased startle reaction to sound, early blindness, seizures, spasticity, and myoclonus. Treatment includes anticonvulsants to control seizures.

For a complete picture of Sandhoff Disease (Jatzkewitz-Pilz Syndrome)’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.