Prader-Willi Syndrome (PWS) Market - Snapshot
Prader–Willi syndrome (PWS) is a genetic disorder caused by a loss of function of specific genes on chromosome 15. In newborns, symptoms include weak muscles, poor feeding, and slow development. Beginning in childhood, those affected become constantly hungry, which often leads to obesity and type 2 diabetes. Mild to moderate intellectual impairment and behavioral problems are also typical of the disorder. Often, affected individuals have a narrow forehead, small hands and feet, short height, light skin and hair. Most are unable to have children. About 74% of cases occur when part of the father's chromosome 15 is deleted. In another 25% of cases, the affected person has two copies of the maternal chromosome 15 from the mother and lacks the paternal copy. As parts of the chromosome from the mother are turned off through imprinting, they end up with no working copies of certain genes. PWS is not generally inherited, but rather the genetic changes happen during the formation of the egg, sperm, or in early development. No risk factors are known for the disorder. Those who have one child with PWS have less than a 1% chance of the next child being affected. A similar mechanism occurs in Angelman syndrome, except the defective chromosome 15 is from the mother, or two copies are from the father.
Prader–Willi syndrome has no cure. Treatment may improve outcomes, especially if carried out early. In newborns, feeding difficulties may be supported with feeding tubes. Strict food supervision is typically required, starting around the age of three, in combination with an exercise program. Growth hormone therapy also improves outcomes. Counseling and medications may help with some behavioral problems. Group homes are often necessary in adulthood.
Prader Willi syndrome (PWS) is a complex genetic disorder results from the lack of expression of genes on the paternally-inherited genes in the15q11.2-q13 chromosome region. The burden of multisystemic complex genetic disorder in patients has been increasingly characterized by massive bearings it has on metabolic, endocrine, and neurologic systems, marred by hypothalamic dysfunction. The patients characteristically suffer from severe hypotonia, particularly neonatal hypotonia, with troubling implications in behavior and intellectual difficulties continuing to later years of life. The condition has led to stunted development. A key drive for PWS market comes from the its prevalence in global populations—estimated as 1/10,000–1/30,000—which is significant irrespective of race, gender, and ethnicities. The key causative factor for the high morbidity and mortality in PWS is obesity.
In developed and developing nations, the rise in uptake in DNA methylation testing has propelled the evolution of the PWS market. In emerging economies, molecular testing with DNA methylation has gained ground in hospital settings. A growing understanding of the etiology and pathophysiology is key to boosting the growth prospects in the market. Policymakers around the world are intent on raising awareness about the burden of the disease to promote early diagnosis and early treatment. These might lead to overall better outcomes, and will offer new opportunities for healthcare players to implement behavioral and cognitive strategies.
Prader-Wili syndrome (PWS) is a rare genetic disorder, which causes problems in intellectual development, behavior, physical growth, mood, and eating. This rare genetic disorder occurs due to changes in chromosome 15, which is responsible for regulating the gene expression. Difficulty to control eating is a major symptom among people with PWS. Other symptoms include moderate to high learning disabilities, possess a compulsive behavior, outbursts, and stubbornness.
Newborns with this disorder may have small hands and feet, almond shaped eyes, and a triangular mouth. Other factors that define PWS included reduced development of genital organs, feeding difficulties, quick weight gain, uncontrollable overeating, and extremely weak muscles. The disorder also causes incomplete or delayed puberty among individuals of both sexes; therefore, they are unable to have children.
In many of the cases, the Prader-Wili syndrome is inherited. However, in some cases the anomaly can take place which can be caused due to malfunctioning of chromosome 15. This may be passed from one generation to the next, especially in maternal uniparental disomy and deletion in paternal chromosome 15. Genetic changes may occur randomly during the formation of reproductive cells or during early development of embryo. In such cases, affected people may or may not have a history of the disorder in their family.
Identify the key factors that will drive your company’s growth, Request a brochure of this report here
Prader-Willi Syndrome (PWS) Market – Competitive Landscape
Chong Kun Dang Pharmaceutical Corp
Founded in 1941, Chong Kun Dang is headquartered in Seoul, South Korea. Company is leading pharmaceutical & biotechnological company in Korea. Company offers range of prescription drugs for anti-diabetics, anticancer, anti-hypertension, health supplements, prebiotics and healthy beverage products. Few of the drugs for Prader-Willi Syndrome (PWS) treatment are under pipeline. Chong Kun Dang Pharmaceutical Corp is the first Korean pharmaceutical company which has got U.S. FDA approval and which export some of the products to the U.S. market.
LG Life Sciences Ltd.
Founded in 2002, LG Life Sciences Ltd. is headquartered in Seoul, South Korea. The company specializes in specialty chemicals in South Korea and international market. Company also offers range of products in animal health products as well as pharmaceuticals. In pharmaceutical segment company offers treatment products for Prader-Wili syndrome, fertility treatment related products, hormones, viscosupplements used during the treatment of degenerative diseases, chemical vaccines etc.
Prader-Willi Syndrome (PWS) Market – Dynamics
Growth hormone continues to be the major constituent of overall therapeutic contribution:-
Currently, pharmacological drugs and therapy for Prader-Willi Syndrome are concentrated primarily on the usage of human growth hormone (HGH) supplementation, which is permitted in the U.S. Supplementation of growth hormone continues is one of the major constituent of overall therapeutic contribution of the market due to nearly pervasive deficiency of growth hormone (GH) in patients suffering from PWS syndrome. Accepted growth hormone therapies in North America and Europe include Genotropin by Pfizer, Nordotropin by Novo Nordisk, and Ominitrope (somatropin injection) from Sandoz. Growth hormone therapies which are increasingly used to treat PWS patients, especially older patients are central nervous system and cardiovascular agents.
Expanding operations in future? To get the perfect launch ask for a custom report here
Clinical Trials Pave the Way for PWS Treatments:-
Increase in investment to find innovative solution for the Prader-Willi syndrome (PWS) by pharmaceutical and biotech companies is expected to drive the market. Many clinical trials are taking place around the world which are helping to better understand syndrome and to develop novice products/ therapy for the treatment of PWS. However, side effects and high cost of these therapies are likely to hinder the global Prader-Willi syndrome market.
Prader-Willi Syndrome (PWS) Market – Segmentation
The Prader-Willi Syndrome (PWS) market can be divided into:
- Product Type
Prader-Willi Syndrome (PWS) Market Segmentation – By Product Type
Depending on the product type, the Prader-Willi Syndrome (PWS) market can be divided into:
- Betahistine Hydrochloride
- Diazoxide Choline CR
Prader-Willi Syndrome (PWS) Market Segmentation – By Application
Depending on the Application, the Prader-Willi Syndrome (PWS) market can be divided into:
This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.
Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:
- Customer Experience Maps
- Insights and Tools based on data-driven research
- Actionable Results to meet all the business priorities
- Strategic Frameworks to boost the growth journey
The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.
The following regional segments are covered comprehensively:
- North America
- Asia Pacific
- Latin America
- The Middle East and Africa
The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.
Below is a snapshot of these quadrants.
1. Customer Experience Map
The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.
2. Insights and Tools
The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.
3. Actionable Results
The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.
4. Strategic Frameworks
The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.
The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:
1. What can be the best investment choices for venturing into new product and service lines?
2. What value propositions should businesses aim at while making new research and development funding?
3. Which regulations will be most helpful for stakeholders to boost their supply chain network?
4. Which regions might see the demand maturing in certain segments in near future?
5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?
6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?
7. Which government regulations might challenge the status of key regional markets?
8. How will the emerging political and economic scenario affect opportunities in key growth areas?
9. What are some of the value-grab opportunities in various segments?
10. What will be the barrier to entry for new players in the market?
Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.