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The report covers emerging Prader-Willi Syndrome drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Prader-Willi Syndrome treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market. 

Prader-Willi Syndrome: An Overview

Prader-Willi syndrome (PWS) is characterized by severe hypotonia, poor appetite, and feeding difficulties in early infancy, followed in early childhood by excessive eating and gradual development of morbid obesity (unless food intake is strictly controlled). Prader-Willi Syndrome is sometimes called Willi-Prader syndrome or Prader-Labhart-Willi syndrome.

The genes associated with Prader-Willi Syndrome normally are expressed only from a region of chromosome 15 inherited from the father. The genes inherited from the mother normally are inactivated. Therefore, children affected with Prader-Willi Syndrome have a deletion or disruption of the chromosome inherited from the father or have inherited two copies of this chromosomal region from the mother. The latter situation is called maternal uniparental disomy.

The symptoms and severity of Prader-Willi Syndrome can vary from one person to another. Many features of the disorder are nonspecific, and others may develop slowly over time or can be subtle. Prenatal diagnosis is possible in families with a previous history of Prader-Willi Syndrome. Prior identification of a disease-causing abnormality can facilitate prenatal testing, but it is available by methylation analysis following amniocentesis regardless of cause.

Treatment of a child affected with PWS involves the primary care physician and a multi-specialty team that includes an ophthalmologist to evaluate for myopia and strabismus, a pediatric endocrinologist for consideration of growth hormone treatment, and a developmental pediatrician.

Prader-Willi Syndrome Market Key Facts

• A study by Ragusa L. et al., (2020), quoted that approximately 65%–70% of Prader-Willi Syndrome cases are due to the deletion of paternal genes on chromosome 15 in the 15q11-q13 region, 20%–30% are caused by maternal uniparental disomy of chromosome 15 and most of the remaining 2%–5% have an imprinting center defect or unbalanced translocations.

• As per the National Organization for Rare Disorders (NORD), Prader-Willi Syndrome affects males and females in equal numbers and occurs in all ethnic groups and geographic regions in the world.

• A study conducted by Gutierrz el al. (2023), to identify the etiology and epidemiology of Prader-Willi Syndrome, medical conditions, and emergencies estimated the prevalence of Prader-Willi Syndrome to be 1 in every 1 in 20000 to 1 in 30000 births.

Prader-Willi Syndrome Market

The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Prader-Willi Syndrome pipeline therapies. It also thoroughly assesses the Prader-Willi Syndrome market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.

The report gives complete details of the market trend for each marketed Prader-Willi Syndrome drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.

Prader-Willi Syndrome Epidemiology Assessment 

The epidemiology section provides insights into the historical, current, and forecasted Prader-Willi Syndrome epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Prader-Willi Syndrome epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders. 

The Report Covers the Prader-Willi Syndrome Epidemiology, Segmented as –

• Total Prevalent Cases of Prader-Willi Syndrome in the 7MM [2019–2032]

• Diagnosed Prevalent Cases of Prader-Willi Syndrome in the 7MM [2019–2032]

• Mutation-specific Diagnosed Prevalent Cases of Prader-Willi Syndrome in the 7MM [2019–2032]

• Treatable Cases of Prader-Willi Syndrome in the 7MM [2019–2032]

Prader-Willi Syndrome Drugs Uptake and Pipeline Development Activities

The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Prader-Willi Syndrome market or expected to be launched during the study period. The analysis covers the Prader-Willi Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Prader-Willi Syndrome drugs based on their sale and market share.

The report also covers the Prader-Willi Syndrome pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Prader-Willi Syndrome companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

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Prader-Willi Syndrome Therapeutics Analysis

Currently, there is no cure for Prader-Willi Syndrome (PWS). The lives of individuals with Prader-Willi Syndrome can be improved with early diagnosis and careful management of symptoms, but more effective therapies are needed.

The treatment of Prader-Willi Syndrome is currently based on treating the symptoms of the disorder as they arise. Growth hormone deficiency is present in almost all children and many adults with Prader-Willi Syndrome. In multiple studies, human growth hormone (HGH) has been found to be beneficial for those with Prader-Willi syndrome.

In June of 2000, HGH was officially approved by the FDA in the United States for use in patients with Prader-Willi syndrome. HGH is effective not only in increasing height, but also in decreasing body fat, increasing muscle mass, improving weight distribution, increasing stamina, and increasing bone mineral density.

Some of the major approved GH therapies include Pfizer’s GENOTROPIN (somatropin) along with other major brands such as NORDITROPIN (somatropin), OMNITROPE (somatropin [rDNA origin] injection) and a few others in the US to treat children with growth failure due to Prader-Willi syndrome.

Several major pharma and biotech companies are developing therapies for Prader-Willi Syndrome. Currently, Soleno Therapeutics is leading the therapeutics with its Prader-Willi Syndrome drug candidates in the most advanced stage of clinical development.

Prader-Willi Syndrome Companies Actively Working in the Therapeutics Market Include

• Soleno Therapeutics

• Levo Therapeutics

• Inversago Pharma

• Saniona

• LG Life Sciences

• GLWL Research

• OptiNose

• Larimar Therapeutics

• Helsinn

• ConSynance Therapeutics

• Neuren Pharmaceuticals

• Radius Health

• Rhythm

• Tonix Pharmaceuticals

And Many Others

Emerging and Marketed Prader-Willi Syndrome Therapies Covered in the Report Include:

• Diazoxide choline controlled release (DCCR): Soleno Therapeutics

• Intranasal carbetocin (LV-101): Levo Therapeutics

And Many More

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Table of Content (TOC)

1. Key Insights

2. Executive Summary 

3. Prader-Willi Syndrome Competitive Intelligence Analysis

4. Prader-Willi Syndrome Market Overview at a Glance

5. Prader-Willi Syndrome Disease Background and Overview

6. Prader-Willi Syndrome Patient Journey

7. Prader-Willi Syndrome Patient Population and Epidemiology Trends (In the US, EU5, and Japan)

8. Prader-Willi Syndrome Treatment Algorithm, Current Treatment, and Medical Practices

9. Prader-Willi Syndrome Unmet Needs

10. Key Endpoints of Prader-Willi Syndrome Treatment

11. Prader-Willi Syndrome Marketed Therapies

12. Prader-Willi Syndrome Emerging Drugs and Latest Therapeutic Advances

13. Prader-Willi Syndrome Seven Major Market Analysis

14. Attribute Analysis

15. Prader-Willi Syndrome Market Outlook (In US, EU5, and Japan)

16. Prader-Willi Syndrome Companies Active in the Market

17. Prader-Willi Syndrome Access and Reimbursement Overview

18. KOL Views on the Prader-Willi Syndrome Market

19. Prader-Willi Syndrome Market Drivers

20. Prader-Willi Syndrome Market Barriers

21. Appendix

22. DelveInsight Capabilities

23. Disclaimer

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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