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 24 drugs in development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) by 23 companies/universities/institutes. The top development phase for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) is preclinical with ten drugs in that stage. The Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) pipeline has 21 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: Sigilon Therapeutics, EdiGene and Neurogt.

The key targets in the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) pipeline products market include Alpha L-Iduronidase (IDUA or EC 3.2.1.76), A Disintegrin And Metalloproteinase With Thrombospondin Motifs 5 (A Disintegrin And Metalloproteinase With Thrombospondin Motifs 11 or Aggrecanase 2 or ADMP2 or ADAMTS5 or EC 3.4.24.), and A Disintegrin And Metalloproteinase With Thrombospondin Motifs 4 (ADMP1 or Aggrecanase 1 or ADAMTS4 or EC 3.4.24.82).

The key mechanisms of action in the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) pipeline product include Alpha L-Iduronidase (IDUA or EC 3.2.1.76) Activator with ten drugs in Phase II. 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.

This content was updated on 25 January 2024

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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.