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.
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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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) and features dormant and discontinued products.
GlobalData tracks 28 drugs in development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) by 17 companies/universities/institutes. The top development phase for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) is preclinical with 14 drugs in that stage. The Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) pipeline has 26 drugs in development by companies and two by universities/ institutes. Some of the companies in the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) pipeline products market are: Phoenix Nest, Denali Therapeutics and Esteve Pharmaceuticals.
The key targets in the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) pipeline products market include Alpha N-Acetylglucosaminidase, N Sulphoglucosamine Sulphohydrolase, and N-Acetylgalactosamine 6 Sulfatase.
The key mechanisms of action in the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) pipeline product include Alpha N-Acetylglucosaminidase Activator with six 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 Fusion Protein.
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.
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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.
