Global Morquio Syndrome Treatment Market to Grow at a CAGR of 7.35% During 2026-2035, Driven by the growing investment in research and development (R&D) in the pharmaceutical sector.

Morquio syndrome, also known as mucopolysaccharidosis type IV (MPS IV), is an uncommon metabolic disease in which the body is unable to break down specific sugar molecules known as glycosaminoglycans. North America holds a significant market portion of the morquio syndrome treatment market share due to the presence of large pharmaceutical companies such as BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), Concert Pharmaceuticals, Inc., and others in the United States and Canada.

In 2022, the U.S. government invested USD 40 million to expand the role of biomanufacturing for active pharmaceutical ingredients (APIs) and antibiotics, leading to product development to partially cure this disease. The National Organisation for Rare Disorders estimates that the disorder affects between 1/40,000 and 1/200,000 newborns. It is estimated that lysosomal storage diseases, MPS IVA (affecting 95% of those with MPS IV), and MPS IVB (affecting 5% of affected persons) affect about one in every 5,000 live births.

The continuous research and development activities support the market growth. In May 2023, Nemours Children’s collaborated with the FNIH AMP® BGTC, a public-private partnership between the FDA, biopharmaceutical and life science companies, U.S. Food and Drug Administration (FDA), non-profits, and other organisations, to help expedite the development and delivery of tailored or ‘bespoke’ gene therapies.

New NIH funding was established in 2021 in order to create a thorough natural history of Morquio A syndrome. In an effort to address this hereditary ailment that results in major side effects such as cervical spinal cord compression, short stature, flat feet, trouble walking, tracheal obstruction, hearing loss, and heart valvular disease, researchers at Nemours have also been investigating novel and cutting-edge therapy approaches.

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Market Analysis by Disease Type, Treatment, Distribution Channels, and Region:

• The market, on the basis of disease type, can be divided into Morquio A and Morquio B.
• Based on treatment, the market can be divided into enzyme replacement therapy and others.
• The various distribution channels for Morquio syndrome treatment include hospital pharmacies, retail pharmacies, and online.
• The major regional markets for Morquio syndrome treatment include North America, Europe, the Asia Pacific, Latin America, and the Middle East and Africa.

Key Findings of the Report:

• The growing investment in research and development (R&D) in the pharmaceutical sector contributes to the Morquio syndrome treatment market expansion. The pharmaceutical industry invested around € 39,600 million in research and development in Europe in 2020.
• According to the World Health Organization (WHO), congenital diseases cause an estimated 240,000 baby deaths globally within 28 days of birth each year. The rising prevalence of congenital diseases is a key market driver to expand research and development on Morquio syndrome.
• The increasing number of products that regulatory bodies have approved can greatly aid in the expansion of the market. Additionally, this offers market players favourable chances to create novel medications, which can further spur the market expansion.
• Treatment procedures such as elosulfase alfa (Vimizim) to cure type A morquio syndrome and advancements in enzyme therapy drive the market growth.

Table: Pharmaceutical Production in Europe, 2021

Key Offerings of the Report:

• The EMR report gives an overview of the global morquio syndrome treatment market for the periods (2019-2025) and (2026-2035).
• The report also offers the historical (2019-2025) and forecast (2026-2035) markets for the disease type, treatment, distribution channels, and regions of Morquio syndrome treatment.
• The report analyses the market dynamics, covering the key demand and price indicators in the market, along with an assessment of the SWOT and Porter’s Five Forces models.

Top Players:

The major players in the global Morquio syndrome treatment market are:

• BioMarin Pharmaceutical Inc 
• JCR Pharmaceuticals Co., Ltd.
• Sanofi
• Sangamo Therapeutics
• Alexion Pharmaceuticals Inc.

The comprehensive report by EMR looks into the market share, capacity, and latest developments like mergers and acquisitions, plant turnarounds, and capacity expansions of the major players.

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