The Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) drugs in development market research report provides comprehensive information on the therapeutics under development for Niemann-Pick Disease Type A (Acid Sphingomyelinase 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 Niemann-Pick Disease Type A (Acid Sphingomyelinase 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 Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) and features dormant and discontinued products.

GlobalData tracks seven drugs in development for Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) by four companies/universities/institutes. The top development phase for Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) is preclinical with five drugs in that stage. The Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) pipeline has five drugs in development by companies and two by universities/ institutes. Some of the companies in the Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) pipeline products market are: Polaryx Therapeutics, IntraBio and Duke University.

The key targets in the Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) pipeline products market include Peroxisome Proliferator Activated Receptor Alpha (Nuclear Receptor Subfamily 1 Group C Member 1 or NR1C1 or PPARA), Retinoic Acid Receptor RXR Alpha (Nuclear Receptor Subfamily 2 Group B Member 1 or Retinoid X Receptor Alpha or NR2B1 or RXRA), and Retinoic Acid Receptor Alpha (RAR Alpha or Nuclear Receptor Subfamily 1 Group B Member 1 or NR1B1 or RARA).

The key mechanisms of action in the Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) pipeline product include Peroxisome Proliferator Activated Receptor Alpha (Nuclear Receptor Subfamily 1 Group C Member 1 or NR1C1 or PPARA) Agonist with three drugs in Preclinical. The Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) pipeline products include two routes of administration with the top ROA being Oral and three key molecule types in the Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) pipeline products market including Small Molecule, and Cell Therapy.

Niemann-Pick Disease Type A (Acid Sphingomyelinase Deficiency) overview

Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disease and an autosomal recessive genetic disorder characterized by the deficiency of sphingomyelinase. It is caused by a mutation of the SMPD1 gene. The subtypes of ASMD include Niemann-Pick disease (NPD) types A and B. Due to the deficiency of sphingomyelinase, sphingomyelin accumulates in the brain, kidneys, liver, and other organs. Enlargement of the liver and spleen, feeding difficulties, loss of reflexes, and cherry red spot macula in infants can be seen in patients with ASMD. It can be diagnosed through molecular genetic testing of the SMPD1 gene. Olipudase alfa (Xenpozyme) enzyme replacement therapy (ERT) helps to reduce the accumulation of sphingomyelin.

For a complete picture of Niemann-Pick Disease Type A (Acid Sphingomyelinase 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.