Summary

Pompe Disease (PD), also known as glycogen storage disease II, is a recessive genetic metabolic disorder caused by a deficiency in the enzyme ?-glucosidase (GAA). GAA is normally responsible for hydrolyzing ?-1, 4 and ?-1, 6 linkages in various carbohydrates, including maltose, isomaltose, and glycogen. The absence or deficiency of GAA results in excessive storage of glycogen in cellular lysosomes. This inhibition can lead to cellular stress and injury. In PD, cardiac muscle tissue is severely affected alongside neurons in both the autonomic nervous system (ANS) and central nervous system (CNS).


Scope

  • GlobalData’s Pompe Disease - Opportunity Assessment and Forecast combines data from the Pharma Intelligence Center with in-house analyst expertise to provide a competitive assessment of the disease marketplace.


    Reasons To Buy
  • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
  • Develop business strategies by understanding the trends shaping and driving the global PD market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global RVO market in the future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.