The Pompe Disease drugs in development market research report provides comprehensive information on the therapeutics under development for Pompe Disease, 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 Pompe Disease. Buy the report here.
Smarter leaders trust GlobalData
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 Pompe Disease and features dormant and discontinued products.
GlobalData tracks 32 drugs in development for Pompe Disease by 30 companies/universities/institutes. The top development phase for Pompe Disease is preclinical with 19 drugs in that stage. The Pompe Disease pipeline has 28 drugs in development by companies and four by universities/ institutes. Some of the companies in the Pompe Disease pipeline products market are: M6P Therapeutics, Amicus Therapeutics and Erasmus MC.
The key targets in the Pompe Disease pipeline products market include Lysosomal Alpha Glucosidase, Glycogen Starch Synthase, and Transferrin Receptor Protein 1.
The key mechanisms of action in the Pompe Disease pipeline product include Lysosomal Alpha Glucosidase Replacement with 12 drugs in Phase III. The Pompe Disease pipeline products include four routes of administration with the top ROA being Intravenous and eight key molecule types in the Pompe Disease pipeline products market including Gene Therapy, and Recombinant Enzyme.
Pompe Disease overview
Pompe disease is an inherited disorder caused by defect in a gene called GAA. The GAA gene is responsible for the production of the GAA enzyme (acid alpha-glucosidase). This enzyme is needed to break down glycogen, a form of sugar stored in muscle cells. When too much glycogen builds up in the muscle cells, the cells become damaged and the muscles cannot function properly. Symptoms include respiratory failure, muscle pain, proximal muscle weakness, respiratory tract infections, headache, and difficulty chewing or jaw muscle fatigue.
For a complete picture of Pompe Disease’s pipeline drug market, buy the report here.
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, 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.
