A recent report published by Infinium Global Research on pompe disease treatment market provides in-depth analysis of segments and sub-segments in the global as well as regional pompe disease treatment market. The study also highlights the impact of drivers, restraints, and macro indicators on the global and regional pompe disease treatment market over the short term as well as long term. The report is a comprehensive presentation of trends, forecast and dollar values of global pompe disease treatment market. According to the report, the global pompe disease treatment market is projected to grow at a CAGR of 9.0% over the forecast period of 2019-2025.

Market Insight

Pompe disease is a rare neuromuscular disorder that occurs in children, infants, and adults who inherit a defective gene from their parents. It is one of the genetic diseases known as lysosomal storage disorders. The symptoms of Pompe disease are insufficient or missing amounts of an enzyme called acid alpha-glucosidase (GAA). This enzyme is needed to break down a complex sugar molecule into glucose.it is a simple sugar that our bodies use for energy. Pompe disease is a progressive disease that gets worse over time. This disease causes progressive muscle weakness and often leads to breathing problems. It is also considered as a metabolic muscle disease. It is similar to other neuromuscular disorders such as muscular dystrophies.

The growing special regulatory drug designations for orphan drugs and increasing research activities performed to find an effective treatment for the disease are driving the pompe disease treatment market. Further Enzyme replacement therapies and growing developments in gene therapy also provide significant growth to the market. However, inflated costs associated with Pompe disease therapy is hindering the growth of the market. Moreover, emerging immunotherapeutic approaches for pompe disease provide growth opportunities for pompe disease treatment market

Geographically, the pompe disease treatment market is segmented into North America, Europe, the Asia-Pacific, and the Row. North America is the largest region for global pompe disease treatment market. The occurrence of the disease is 1 out of 40,000 births in the U.S. drug and food administration has approved an enzyme replacement therapy Lumizyme® is used for patients older than 8 years of age. Whereas Myozyme® for the infantile-onset of Pompe disease suffering patients of less than 8 years old. Asia-Pacific is expected to grow with healthy CAGR owing to healthcare facilities and increasing awareness among patients regarding rare disorders.

Segment Covered

The report on global pompe disease treatment market covers segments such as therapy type, molecule type, route of administration, and dosage forms. On the basis of therapy type, the sub-markets include enzyme replacement therapy, and substrate reduction therapy. On the basis of molecule type, the sub-markets include biologics, and small molecules. On the basis of route of administration, the sub-markets include oral, and parenteral. On the basis of dosage forms, the sub-markets include solid, and liquid.

Companies Profiled:

The report provides profiles of the companies in the market such as BioMarin Pharmaceutical, Amicus Therapeutics, Genzyme company, Oxyrane, Audentes Therapeutics, Sangamo BioSciences, EpiVax, and Valerion Therapeutics.

Report Highlights:

The report provides deep insights into the demand forecasts, market trends, and micro and macro indicators. In addition, this report provides insights into the factors that are driving and restraining the growth in this market. Moreover, The IGR-Growth Matrix analysis given in the report brings an insight into the investment areas that existing or new market players can consider. The report provides insights into the market using analytical tools such as Porter's five forces analysis and DRO analysis of pompe disease treatment market. Moreover, the study highlights current market trends and provides forecast from 2019-2025. We also have highlighted future trends in the market that will affect the demand during the forecast period. Moreover, the competitive analysis given in each regional market brings an insight into the market share of the leading players.