Press Release
Press Release
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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Table: Pharmaceutical Production in Europe, 2021
The major players in the global Morquio syndrome treatment market are BioMarin Pharmaceutical Inc., JCR Pharmaceuticals Co., Ltd., Sanofi, Sangamo Therapeutics, and Alexion Pharmaceuticals Inc., among others. 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.
REPORT FEATURES | DETAILS |
Base Year | 2023 |
Historical Period | 2017-2023 |
Forecast Period | 2024-2032 |
Scope of the Report |
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
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Breakup by Disease Type |
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Breakup by Treatment |
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Breakup by Distribution Channels |
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Breakup by Region |
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Market Dynamics |
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Competitive Landscape |
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Companies Covered |
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