Achondroplasia: Introduction

  • Achondroplasia is a genetic disorder characterized by underdevelopment of bones, particularly the long bones of the body leading to dwarfism. The word ‘achondroplasia’ literally means ‘without cartilage formation’. However, the problem with the condition is not with cartilage formation, but with conversion of cartilage to bones, a process called ossification. Achondroplasia is caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene that results in its protein being overactive. The disorder has an autosomal dominant mode of inheritance; meaning only one mutated copy of the gene is required for the condition to occur. Achondroplasia is also the most common form of dwarfism. The condition is known to affect 1 in 15,000 to 40,000 newborns.
  • Achondroplasia is characterized by short arms and legs, but typically a normal length torso. The average adult height of those affected ranges from 4 ft to 4.4 ft. Other common features include short arms and legs, average-size trunk, an enlarged head, and a prominent or protruding forehead. The complications or risk factors associated include breathing difficulties such as sleep apnea, obesity, spinal stenosis, and recurrent ear infections. The disorder does not generally affect intelligence.

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Key Drivers and Restraints of Global Achondroplasia Treatment Market

  • Currently, there is no cure or prevention for achondroplasia. However, growth hormone therapy and surgery is used to minimize the conditions and help the patient to have apparently normal growth and life. Other supportive treatments are used to treat or prevent complications such as obesity, hydrocephalus, obstructive sleep apnea, middle ear infections, or spinal stenosis.
  • Rise in awareness about timely diagnosis of congenital disorders is projected to propel the global achondroplasia treatment market during the forecast period. Rise in cases of achondroplasia results in increase in research & development by companies and research institutes in clinical trials to manufacture novel therapeutics. Increase in investment in research & development for the treatment and management of achondroplasia is expected to drive the global market during the forecast period.
  • However, poor reimbursement, lack of awareness, and short duration effectiveness of growth hormone therapy is anticipated to hamper market growth

Supportive Therapy to Lead Global Market

  • The global achondroplasia treatment market can be segmented based on treatment type, end-user, and region
  • In terms of treatment type, the global achondroplasia treatment market can be divided into growth hormone therapy, surgery, and supportive therapy. Supportive therapy is projected to be the fastest growing segment of the global achondroplasia treatment market.
  • Based on end-user, the global achondroplasia treatment market can be classified into hospitals, specialty clinics, retail pharmacies, and others

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Asia Pacific to be Lucrative Market

  • The global achondroplasia treatment market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa
  • Improving health care infrastructure, rise in awareness about timely diagnosis of underlying disorders, and large undiagnosed patient population are anticipated to present significant opportunities in the achondroplasia treatment market in Asia Pacific in the near future. Increase in public and private health care expenditure in countries such as China, India, and Brazil is expected to attract manufacturers toward these markets.

Key Players Operating in Global Achondroplasia Treatment Market

Leading players in the global achondroplasia treatment market include:

  • Ascendis Pharma A/S
  • BioMarin Pharmaceutical, Inc.
  • Ribomic, Inc.

Global Achondroplasia Treatment Market: Research Scope

Global Achondroplasia Treatment Market, by Treatment Type

  • Growth Hormone Therapy
  • Surgery
  • Supportive Therapy

Global Achondroplasia Treatment Market, by End-user

  • Hospitals
  • Specialty Clinics
  • Retail Pharmacies
  • Others

Global Achondroplasia Treatment Market, by Region

  • North America
    • U.S.
    • Canada
  • Europe
    • Germany
    • U.K.
    • France
    • Italy
    • Spain
    • Rest of Europe
  • Asia Pacific
    • China
    • India
    • Japan
    • Australia & New Zealand
    • Rest of Asia Pacific
  • Latin America
    • Brazil
    • Mexico
    • 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
  • Europe
  • 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.

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Achondroplasia Treatment Market

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