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
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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.
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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.
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