Overview and Scope
Mucolipidosis II or I-cell disease, is a rare inherited condition where cells cannot properly break down certain substances, causing organs and tissues to deteriorate over time. Mucolipidosis II results from enzyme mutations that impair cellular substance breakdown, causing skeletal abnormalities and developmental delays in patients.
Sizing and Forecast
The mucolipidosis II market size has grown steadily in recent years. It will grow from $12.61 billion in 2023 to $13.13 billion in 2024 at a compound annual growth rate (CAGR) of 4.1%. The growth in the historic period can be attributed to increasing regulatory approvals for orphan drugs, rise in awareness for mucolipidosis II disease, surge in clinical trials, increasing genetic testing, increasing funding for rare disease research, and increasing diagnosis.
The mucolipidosis II market size is expected to see steady growth in the next few years. It will grow to $15.52 billion in 2028 at a compound annual growth rate (CAGR) of 4.3%. The growth in the forecast period can be attributed to increasing awareness among healthcare professionals, rise in research and development activities, emerging biomarkers for early diagnosis, growing investment in rare disease therapies, increasing reimbursement policies, and increasing demand of gene therapy. Major trends in the forecast period include advancements in artificial intelligence in medicine, advancements in genetic testing technologies, advancements in diagnostic methods, advancements in therapeutic treatments, and advancements in telemedicine.
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Segmentation & Regional Insights
The mucolipidosis ii market covered in this report is segmented –
1) By Treatment: Antibiotics, Physical Therapy, Hip Replacement, Experimental Therapies, Other Treatments
2) By Mode Of Administration: Injectable, Oral, Other Modes Of Administration
3) By Symptoms: Deafness, Lack Of Muscle Tone (Hypotonia), Abnormal Spine Curvature, Changing Proportion of Mental Retardation, Low Growth Of Gross And Fine Motor Skills, Other Symptoms
4) By End-User: Hospitals, Homecare, Specialty Clinics, Other End-Users
North America was the largest region in the mucolipidosis II market in 2023. The regions covered in the mucolipidosis ii market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.
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Major Driver Impacting Market Growth
The rise in gene therapy is expected to propel the growth of the mucolipidosis II market going forward. Gene therapy is an advanced medical technique that aims to treat or prevent genetic disorders by correcting faulty genes or introducing new genes into a patient’s cells through methods such as gene addition, gene editing, gene silencing, and gene regulation, gene therapy. Gene therapy offers a promising approach to treating ML II by addressing the root genetic cause of the disorder. The approach involves introducing a functional copy of the gene into the patient’s cells to restore the production of the enzyme. For instance, in April 2024, according to the American Society of Gene & Cell Therapy, a US-based organization for gene and cell therapy, the number of gene therapies in phase III clinical trials increased by 10% in the third quarter of 2023, marking the first quarterly rise since the third quarter of 2022. Therefore, the rise in gene therapy will drive the market for mucolipidosis II.
Impact of Increased Research and Development Activities on the Mucolipidosis II Market
The rise in research and development activities is expected to propel the growth of the mucolipidosis II market going forward. Research and development (R&D) activities involve systematic investigation and experimentation to discover new knowledge or create new products, processes, or services. Research and development (R&D) activities are increasing due to the growing need for innovation and competitive advantage in various industries. Research and development activities are crucial for advancing treatments for Mucolipidosis II by improving the understanding of the disease, developing gene therapies to address underlying genetic mutations, enhancing delivery methods, and conducting preclinical and clinical trials. For instance, in April 2024, according to the Office for National Statistics, a UK-based government department for collecting and publishing statistics, in 2022, the UK government’s spending on research and development increased to £15.5 ($19.67) billion from £14.0 ($17.77) billion in 2021, marking a 10.5% rise. Therefore, the rise in research and development activities will drive the market for mucolipidosis II.
Key Industry Players
Major companies operating in the mucolipidosis ii market are Pfizer Inc., Merck & Co. Inc., Sanofi S.A., Novartis AG, Takeda Pharmaceutical Company Limited, Teva Pharmaceutical Industries Ltd., Sun Pharmaceutical Industries Limited, Jazz Pharmaceuticals, Cipla Limited, Lupin Limited, Zydus Lifesciences Limited, Alembic Pharmaceuticals, Ultragenyx Pharmaceutical Inc., Regenxbio Inc., Denali Therapeutics Inc., Mylan N.V., Intellia Therapeutics Inc., Avrobio Inc., Orchard Therapeutics plc, Eloxx Pharmaceuticals Inc., Lysogene S.A., Sio Gene Therapies, Homology Medicines Inc., BSN medical GmbH, Abeona Therapeutics Inc
The mucolipidosis ii market report table of contents includes:
- Executive Summary
- Mucolipidosis II Market Characteristics
- Mucolipidosis II Market Trends And Strategies
- Mucolipidosis II Market – Macro Economic Scenario
- Global Mucolipidosis II Market Size and Growth
…………….
- Global Mucolipidosis II Market Competitive Dashboard
- Key Mergers And Acquisitions In The Mucolipidosis II Market
- Mucolipidosis II Market Future Outlook and Potential Analysis
- Appendix
List Of Tables:
Table 1: Global Historic Market Growth, 2018-2023, $ Billion
Table 2: Global Forecast Market Growth, 2023-2028F, 2033F, $ Billion
Table 3: Global Mucolipidosis II Market, Segmentation By Treatment, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
Table 4: Global Mucolipidosis II Market, Segmentation By Mode Of Administration, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
Table 5: Global Mucolipidosis II Market, Segmentation By Symptoms, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
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Table 75: Pfizer Inc. Financial Performance
Table 76: Merck & Co. Inc. Financial Performance
Table 77: Sanofi S.A. Financial Performance
Table 78: Novartis AG Financial Performance
Table 79: Takeda Pharmaceutical Company Limited Financial Performance
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