Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment: Introduction
- Hutchinson-Gilford progeria syndrome (HGPS), is a rare, fatal, genetic condition in children with striking features resembling premature aging. Children with progeria usually have a normal appearance in early infancy.
- At approximately nine to 24 months of age, affected children begin to experience profound growth delays, resulting in short stature and low weight.
- They also develop a distinct facial appearance, characterized by a disproportionately small face in comparison to the head, an underdeveloped jaw, malformation and crowding of the teeth, abnormally prominent eyes, a small nose, and a subtle blueness around the mouth.
- Furthermore, by the second year of life, the scalp hair, eyebrows, and eyelashes are lost (alopecia), and the scalp hair may be replaced by small, downy, white or blond hair
Key Drivers of Global Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment Market
- The global HGPS treatment market is anticipated to expand rapidly in the near few years. Introduction of novel therapies and increase in cases of HGPS are likely to be major drivers of the Hutchinson-Gilford Progeria Syndrome (HGPS) treatment market.
- According to the National Organization for Rare Disorders (NORD), the prevalence of HGPS is approximately 1 in 20 million; therefore, at any given time, approximately 400 children are living with progeria worldwide.
- Use of genetic testing has been increasing substantially in the last few years, thereby positively influencing the genetic disease diagnosis market
- In November, 2020 the U.S. Food and Drug Administration approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older; the first-ever treatment for Progeria. Zokinvy, a farnesyltransferase inhibitor, is an oral medication that helps prevent the buildup of defective progerin or progerin-like protein.
Hospital Pharmacies to be Highly Lucrative Segment
- Based on distribution channel, the global Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment market can be categorized into retail pharmacies, hospital pharmacies, online pharmacies, and others
- The hospitals segment is anticipated to account for a major share of the market in the next few years due to a rise in the number of treatments in hospitals
North America to Offer Significant Opportunities in Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment Market
- In terms of region, the global Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa
- North America accounted for a significant share of the global market in 2020, owing to favorable healthcare infrastructure, high per capita income, and high rate of adoption of advanced surgeries in the region
- Meanwhile, Asia Pacific is expected to offer significant opportunity for the market in the next few years. Significant expansion of the market in Asia Pacific is attributed to increase in healthcare expenditure, surge in government efforts to support medical tourism, and rapid advances made in the biotechnology industry. Increase in healthcare spending by a large section of people in emerging nations, such as India and China, is expected to further fuel the market in the region during the forecast period.
Key Players Operating in Global Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment Market
The global Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment market is consolidated, with the presence of international and local players. Key players operating in the global Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment market are:
- Eiger BioPharmaceuticals
- Teva Pharmaceutical, Inc.
- Novartis AG
Global Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment Market: Research Scope
Global Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment Market, by End-user
- Specialty Clinics
Global Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment Market, by Region
- North America
- Rest of Europe
- Asia Pacific
- Australia & New Zealand
- Rest of Asia Pacific
- Latin America
- Rest of Latin America
- Middle East & Africa
- GCC Countries
- South Africa
- Rest of Middle East & Africa
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.