- 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.
Are you a Start-up willing to make it Big in the Business? Grab an Exclusive PDF Brochure of Achondroplasia Treatment Market Report
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
Stuck in a Neck-to-Neck Competition with Other Brands? Request a Custom Report on “Achondroplasia Treatment Market” here
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
- Supportive Therapy
Global Achondroplasia Treatment Market, by End-user
- Specialty Clinics
- Retail Pharmacies
Global Achondroplasia 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 upcoming business intelligence report is intended to be a one-stop market research solution to guide organizations in taking timely and effective decisions for their business growth. The ongoing study is the culmination of our deep expertise on data science methods, combined with an unparalleled understanding of the external environment and analysis of the industries. Each of the reports by TMR aims at offering a detailed scrutiny of the value chain of our current and prospective customers, takes them from identifying value propositions, and subsequently assist their business executives to implement right strategies to create customer value. We believe that creating and sustaining the customer value is the key pivot on which business executives can create profits for their organizations and increase the market shares, continuously and with sustainability.
Each of our touchpoints through which we engage with the organization/enterprise during the primary research enables us to understand the internal environment it operates in and the ways in which the external environment influences its growth.
Always Evolving with Competition
Excellence is built into the DNA of TMR, and all our teams work collaboratively and committedly toward achieving that in whatever they do. From the very first interaction our teams of research analysts have with any interested market participant to the final preparation processes in each of our reports reflects a rigorous understanding of the dynamic industry environment that businesses operate in.
We relentlessly work to be a differentiator in whatever we do in the current era of hypercompetitive global environment. The result of our unceasing focus on excellence is reflected clearly in the insights and guidance we offer to our customers in order to empower them to create brand equity. Our simple yet very powerful underpinning for navigating the competition is to always keep evolving with changing business environments. Our close-kinit team of research analysts not only believe in this simple philosophy but also implement the same.
Constantly Innovating Our Research Methodologies and Models
We do not limit with any specific market research tool or one standard approach for obtaining or analyzing data. Undoubtedly, our report making process is ever-evolving to meet the customer needs and demands supported broadly by a vast knowledge of data science methods that we combine with business acumen our teams have built upon for several years. Evidently, our primary and secondary research data about customers and consumers have shown that we can go to any lengths to obtain them. This is followed by applying next-gen automation tools integrated with human judgement to clear the clutter, ascertain the most relevant and recent qualitative and quantitative insights, and package them in digestible chunks in our reports for our customers.
We Take Pride in No-One-Size-Fits-All Approach
Over the past several years, we have been conducting market intelligence studies across all industries for all types of organizations — big and small, profit and not-for-profit, and Fortune 500 and Unicorns, as we believe that a sound decision making must be based on no-one-size-fits-all approach. Without doubt, each of the organizations regardless of the industry they are in are affected differently by changes in political, economic, socio-culture, legal, ecological, and legal environments. The research models and the value chain analysis process that our analysts employ for conducting the study uphold this approach.
We look at myriad components of internal and external business environment affecting the growth strategies of organizations. Some of the broader aspects that we focus on are:
- Our team of Analysts and Support Teams always strive to see the bigger picture in life cycle stages of any industry
- The Teams focus on obtaining valuable insights into different models of competitive advantage while making an internal environment analysis
- They keep on modifying the value chain analysis processes of organizations to understand on how customer value is created
Some areas of assessing market dynamics that are used in the research models and methodologies adopted by our analysts can be summarized as:
- Corporate and business strategies underlying new brand positioning strategies
- Mapping for strategic planning for business units
- Various metrics for business portfolio analysis, including BCG matrix
- Value chain analysis
These will help create and sustain competitive advantage for our prospects and current customers.
Key focus areas and agenda that underpin and catalyze our primary and secondary research initiatives and endeavors are:
- What are some of the core competencies and distinctive competencies of new entrants and established players in the industry we are analyzing?
- What are some of the branding opportunities that are emerging at a rapid pace?
- What are the major competitive forces and elements of external environment shaping the industry life cycle stages of most organizations?
- Why some competitive strategies are popular than others among leaders in a certain market?
- What customer-based brand equity strategies companies have leveraged in a certain industry or set of related industries in a sector?
- What are some of the key pricing strategies that companies in an industry are implementing for marketing their products internationally?
- Identifying whether market-push or market-pull strategies are more relevant to a certain product or a service?
Disclaimer: This ongoing market research study is a meticulously planned activity where a broad array of factors and aspects that shape the marketing environment and industry are taken into account. However, keeping in mind the constantly changing nature of business dynamics and changing strategic intents, we are always actively making iterations and modifications in our approaches. As always, we are ready to tailor our insights and guidance to suit your requirements. Engage with us to know what more is there for your CXOs.