The Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) drugs in development market research report provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (MPS I) (Hurler 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 Mucopolysaccharidosis I (MPS I) (Hurler 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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) and features dormant and discontinued products.
GlobalData tracks 20 drugs in development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) by 20 companies/universities/institutes. The top development phase for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) is preclinical with eight drugs in that stage. The Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) pipeline has 17 drugs in development by companies and three by universities/ institutes. Some of the companies in the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) pipeline products market are: RegenxBio, University of Alabama and Mina Therapeutics.
The key targets in the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) pipeline products market include Alpha L-Iduronidase, A Disintegrin And Metalloproteinase With Thrombospondin Motifs 5, and A Disintegrin And Metalloproteinase With Thrombospondin Motifs 4.
The key mechanisms of action in the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) pipeline product include Alpha L-Iduronidase Replacement with eight drugs in Phase III. The Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) pipeline products include nine routes of administration with the top ROA being Intravenous and eight key molecule types in the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) pipeline products market including Gene Therapy, and Small Molecule.
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) overview
Mucopolysaccharidosis type I (MPS I) is an inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness, and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy, and gene therapy.
For a complete picture of Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )’s pipeline drug market, buy the report here.
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