The Niemann-Pick Disease drugs in development market research report provides comprehensive information on the therapeutics under development for Niemann-Pick Disease, 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 Niemann-Pick Disease. 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 Niemann-Pick Disease and features dormant and discontinued products.
GlobalData tracks 34 drugs in development for Niemann-Pick Disease by 30 companies/universities/institutes. The top development phase for Niemann-Pick Disease is preclinical with 22 drugs in that stage. The Niemann-Pick Disease pipeline has 27 drugs in development by companies and seven by universities/ institutes. Some of the companies in the Niemann-Pick Disease pipeline products market are: IntraBio, Scenic Biotech and Polaryx Therapeutics.
The key targets in the Niemann-Pick Disease pipeline products market include NPC Intracellular Cholesterol Transporter 1, Sphingomyelin Phosphodiesterase, and Peroxisome Proliferator Activated Receptor Alpha.
The key mechanisms of action in the Niemann-Pick Disease pipeline product include NPC Intracellular Cholesterol Transporter 1 Activator with five drugs in Preclinical. The Niemann-Pick Disease pipeline products include seven routes of administration with the top ROA being Oral and nine key molecule types in the Niemann-Pick Disease pipeline products market including Small Molecule, and Gene Therapy.
Niemann-Pick Disease overview
Acid sphingomyelinase deficiency (ASMD) is a rare genetic disorder that affects the metabolism of a lipid called sphingomyelin. ASMD is caused by mutations in the SMPD1 gene, which encodes the enzyme acid sphingomyelinase (ASM). ASM is responsible for breaking down sphingomyelin in the lysosomes of cells. When ASM is deficient, sphingomyelin accumulates in various tissues and organs, causing damage and dysfunction. ASMD is also known as Niemann-Pick disease types A and B, depending on the severity and presence of neurological symptoms. ASMD can cause hepatosplenomegaly, thrombocytopenia, ataxia, dystonia, dementia, and bone abnormalities. ASMD is inherited in an autosomal recessive manner and has no cure. Treatment is supportive and symptomatic.
For a complete picture of Niemann-Pick Disease’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.
