Morquio Syndrome Market

Market Overview:

The 7 major Morquio syndrome markets are expected to exhibit a CAGR of 4.45% during 2024-2034.

The Morquio syndrome market has been comprehensively analyzed in IMARC's new report titled "Morquio Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Morquio syndrome, also known as mucopolysaccharidosis type IV (MPS IV), refers to a rare genetic disorder caused by the deficiency or malfunctioning of enzymes involved in the breakdown of certain sugar molecules called glycosaminoglycans (GAGs). The most prominent symptoms associated with the ailment are short stature, skeletal abnormalities, joint laxity, pectus carinatum (protruding chest), hip dysplasia, etc. Various respiratory complications, like obstructive sleep apnea and restrictive lung disease, may also occur. Some other common indications include vision and hearing impairment, cardiac issues, dental problems, etc. The diagnosis of the ailment involves a combination of clinical evaluation, biochemical testing, genetic testing, and imaging studies. Clinical assessment may consist of a physical examination, evaluation of medical history, as well as identification of characteristic symptoms. Several biochemical tests, such as measuring GAGs or enzyme activity levels in blood or urine, aid in confirming the diagnosis. Genetic testing plays a crucial role in identifying specific gene mutations associated with Morquio syndrome. Additionally, numerous imaging studies, including X-rays, CT scans, MRIs, etc., help to visualize skeletal abnormalities and organ involvement.

The increasing cases of deficiency or malfunctioning of specific enzymes, such as N-acetylgalactosamine-6-sulfatase and beta-galactosidase, that disrupt normal tissue functioning are primarily driving the Morquio syndrome market. Furthermore, the rising adoption of effective pain management strategies, including physical therapy, occupational therapy, medications, etc., to help alleviate pain and improve daily functioning in patients is acting as another significant growth-inducing factor. Additionally, the emerging popularity of enzyme replacement therapy (ERT), which involves administering a synthetic form of the deficient enzyme to replace the missing or dysfunctional enzyme responsible for breaking down GAGs, is also creating a positive outlook for the market. Moreover, the increasing utilization of orthopedic interventions, such as corrective surgeries and spinal fusion, for addressing the skeletal issues associated with the ailment, including short stature, skeletal deformities, joint abnormalities, etc., is further augmenting the market growth. Apart from this, the ongoing advancements in biomarker analysis techniques, such as the introduction of liquid chromatography-mass spectrometry (LC-MS) and enzyme-linked immunosorbent assay (ELISA), which have improved the accuracy and sensitivity of biomarker detection, are expected to drive the Morquio syndrome market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the Morquio syndrome market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for Morquio syndrome and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Morquio syndrome market in any manner.

Key Highlights:

• Morquio syndrome is a rare genetic birth condition that is predicted to affect one out of every 200,000 newborns.
• The majority of instances, almost two-thirds, manifest as new mutations.
• Morquio syndrome is predicted to affect one in every 200,000 to 300,000 live births in the United States.
• According to the Children's Hospital of Philadelphia, Morquio syndrome is classified into two types: A and B.
• The condition may not be obvious at birth; symptoms typically develop between the ages of one and three.

Drugs:

VIMIZIM is a prescription drug recommended for people with mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). This drug contains the enzyme elosulfase alfa. Elosulfase alfa is a recombinant human enzyme generated through genetic engineering in Chinese Hamster Ovary (CHO) cells. It replaces the native enzyme in patients with MPS IVA.

Time Period of the Study

• Base Year: 2023
• Historical Period: 2018-2023
• Market Forecast: 2024-2034

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan
   

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the Morquio syndrome market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the Morquio syndrome market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current Morquio syndrome marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the Morquio syndrome market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
• What was the country-wise size of the Morquio syndrome market across the seven major markets in 2023 and what will it look like in 2034?
• What is the growth rate of the Morquio syndrome market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?
   

Epidemiology Insights

• What is the number of prevalent cases (2018-2034) of Morquio syndrome across the seven major markets?
• What is the number of prevalent cases (2018-2034) of Morquio syndrome by age across the seven major markets?
• What is the number of prevalent cases (2018-2034) of Morquio syndrome by gender across the seven major markets?
• How many patients are diagnosed (2018-2034) with Morquio syndrome across the seven major markets?
• What is the size of the Morquio syndrome patient pool (2018-2023) across the seven major markets?
• What would be the forecasted patient pool (2024-2034) across the seven major markets?
• What are the key factors driving the epidemiological trend of Morquio syndrome?
• What will be the growth rate of patients across the seven major markets?
   

Morquio Syndrome: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for Morquio syndrome drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Morquio syndrome market?
• What are the key regulatory events related to the Morquio syndrome market?
• What is the structure of clinical trial landscape by status related to the Morquio syndrome market?
• What is the structure of clinical trial landscape by phase related to the Morquio syndrome market?
• What is the structure of clinical trial landscape by route of administration related to the Morquio syndrome market?