(PharmaNewsWire.Com, December 05, 2018 ) Pompe Disease-Disease Understanding and Treatment Algorithm
Pompe Disease (also known as glycogen storage disease type II [GSDII], acid maltase deficiency or GAA deficiency) is an autosomal recessive inherited disorder caused by a mutation in the gene encoding GAA. Deficiency in GAA affects lysosomal-mediated degradation of glycogenesis and results in intralysosomal accumulation of glycogen and disruption of the tissue architecture in various organs, most notably the skeletal muscles, heart and liver.
The Publisher Pompe Disease market report gives the thorough understanding of the Pompe Disease by including details such as disease definition, classification, symptoms, etiology, pathophysiology, diagnostic trends. It also provides treatment algorithms and treatment guidelines for Pompe Disease in the US, Europe, and Japan.
The Pompe Disease epidemiology division provide the insights about historical and current patient pool and forecasted trend for every 7 major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the Publisher report also provides the diagnosed patient pool and their trends along with assumptions undertaken.
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology scenario of Pompe Disease in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom) and Japan from 2016-2027.
According to Clinical Therapeutics-Genetics and epidemiology of Pompe disease, the overall worldwide incidence of Pompe Disease is usually estimated to be 1:40,000 live births with the late-onset form of the disease generally being more common than the early neonatal form. According to US National Library of Medicine National Institutes of Health, the prevalence of Pompe disease is approximately 1 in 28,000 in the United States.
Pompe Disease Drug Chapters
This segment of the Pompe Disease report encloses the detailed analysis of marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.
The treatment of Pompe disease is disease-specific, symptomatic, and supportive. Enzyme replacement therapy is an approved treatment for all patients with Pompe disease. Myozyme (alglucosidase alfa), the first and only enzyme replacement therapy (ERT) to treat the underlying cause of Pompe disease, has been approved for use in several countries around the world. Apart from ERT, alternative treatments for Pompe disease, such as gene therapy and substrate reduction therapy can also be considered as an attractive option in future.
The Pompe Disease market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.
This segment gives a through detail of market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
Key players such as Sanofi, Amicus Therapeutics, Valerion Therapeutics and many others are involved in developing therapies for Pompe Disease. Expected launch of emerging therapies such as GZ402666 (Sanofi), AT-2220 (Amicus Therapeutics), and other compelling treatments, are expected to impact the market size of Pompe Disease in upcoming years.
Pompe Disease Drugs Uptake
This section focusses on the rate of uptake of the potential drugs recently launched in the market or will get launched in the market during the study period from 2016-2027. The analysis covers market uptake by drugs; patient uptake by therapies and sales of each drug.
This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allows the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
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