The Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) drugs in development market research report provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome ) and features dormant and discontinued products.

GlobalData tracks 32 drugs in development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) by 20 companies/universities/institutes. The top development phase for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) is preclinical with 19 drugs in that stage. The Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) pipeline has 31 drugs in development by companies and one by universities/ institutes. Some of the companies in the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) pipeline products market are: Phoenix Nest, Denali Therapeutics and Amicus Therapeutics.

The key targets in the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) pipeline products market include Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50), N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1), and N-Acetylgalactosamine 6 Sulfatase (Chondroitinsulfatase or Chondroitinase or Galactose 6 Sulfate Sulfatase or N Acetylgalactosamine 6 Sulfate Sulfatase or GALNS or EC 3.1.6.4).

The key mechanisms of action in the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) pipeline product include Alpha N Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) Activator with seven drugs in Preclinical. The Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) pipeline products include ten routes of administration with the top ROA being Intravenous and eight key molecule types in the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) pipeline products market including Gene Therapy, and Recombinant Enzyme.

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) overview

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is an inherited metabolic disease caused by an absence or malfunctioning of certain enzymes needed to break down molecules called glycosaminoglycans (GAGs). Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation, and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT).

For a complete picture of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome )’s pipeline drug market, buy the report here.

This content was updated on 25 January 2024

Data Insights

From

The gold standard of business intelligence.

Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.

GlobalData

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

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.