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Lysosomal Disease Treatment Market Expansion Continues, with Forecast Valuation of $13.7 Billion by 2029

07-08-2025 07:46 AM CET | Health & Medicine

Press release from: The Business Research Company

Lysosomal Disease Treatment Market

Lysosomal Disease Treatment Market

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Lysosomal Disease Treatment Market Size Valuation Forecast: What Will the Market Be Worth by 2025?
The market for treating lysosomal diseases has seen robust growth in recent years. The market size, which stands at $9.22 billion in 2024, is projected to soar to $9.88 billion in 2025, with a compound annual growth rate (CAGR) of 7.2%. Factors contributing to growth during the historical period include advancements in therapeutic strategies, better comprehension and knowledge, increased clinical trials and research efforts, progress in diagnostic tools, and the establishment of neonatal screening programs.

Lysosomal Disease Treatment Market Size Forecast: What's the Projected Valuation by 2029?
In the coming years, the market size of treatments for lysosomal diseases is predicted to experience substantial growth, projected to reach "$13.7 billion by 2029 with a calculated compound annual growth rate (CAGR) of 8.5%. This growth during the forecast period is attributed to the advancements in gene therapy, ongoing progress in enzyme replacement therapy (ERT), the introduction of new therapeutic methods, improved screening and early diagnosis schemes, and increased investment in research and development. The forecast period is expected to see trends such as advancements in enzyme replacement therapy (ERT), innovations in gene therapy, the growth of chaperone therapy, substrate reduction therapy (SRT), and the extension of newborn screening programs.

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What Are the Drivers Transforming the Lysosomal Disease Treatment Market?
The advancement of the lysosomal disease treatment market is anticipated to be driven by escalating investments in healthcare infrastructure. This refers to the assignment of financial means to the progress, preservation, and enhancement of healthcare facilities, tools, and services. In the context of lysosomal disease treatment, an increase in these investments can substantially improve patient outcomes, expedite research advancements, and heighten the overall efficiency of healthcare. For instance, The Moscow Times, an online newspaper based in Russia, reported that Russia's federal project aimed at the modernization of primary healthcare is slated to allocate 88.8 billion rubles ($910 million) in 2023, and 550 billion rubles ($5.7 billion) for the period of 2023-25. As a result of these strengthening investments, the lysosomal disease treatment market is experiencing growth.

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What Long-Term Trends Will Define the Future of the Lysosomal Disease Treatment Market?
Dual-component therapies are gaining traction in the lysosomal disease treatment market. In September 2023, US-based Amicus Therapeutics received FDA approval for Pombiliti and Opfolda, a combined therapy targeting late-onset Pompe disease to improve muscle strength and respiratory function in patients unresponsive to prior treatments.

Which Segments in the Lysosomal Disease Treatment Market Offer the Most Profit Potential?
The lysosomal disease treatment market covered in this report is segmented -

1) By Disease Type: Mucopolysaccharidosis, Pompe Syndrome, Fabry Diseases, Gaucher's Disease, Other Disease Types
2) By Therapy: Substrate Reduction Therapy, Stem Cell Therapy, Enzyme Replacement Therapy, Other Therapies
3) By Route Of Administration: Oral, Parenteral, Other Routes Of Administration
4) By End User: Hospitals, Specialty Clinics, Homecare, Other End Users

Subsegments:
1) By Mucopolysaccharidosis: MPS I (Hurler Syndrome), MPS II (Hunter Syndrome), MPS III (Sanfilippo Syndrome), MPS IV (Morquio Syndrome), MPS VI (Maroteaux-Lamy Syndrome), MPS VII (Sly Syndrome)
2) By Pompe Syndrome: Late-Onset Pompe Disease, Infantile-Onset Pompe Disease
3) By Fabry Disease: Classic Fabry Disease, Late-Onset Fabry Disease
4) By Gaucher's Disease: Type 1 Gaucher Disease, Type 2 Gaucher Disease, Type 3 Gaucher Disease
5) Other Disease Types: Niemann-Pick Disease, Krabbe Disease, Tay-Sachs Disease

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Which Firms Dominate the Lysosomal Disease Treatment Market by Market Share and Revenue in 2025?
Major companies operating in the lysosomal disease treatment market include Eli Lilly and Company, Takeda Pharmaceutical Company Limited, Sanofi SA, Pfizer Inc., Novartis International AG, Johnson & Johnson Services Inc., Alexion Pharmaceuticals Inc., Amicus Therapeutics Inc., Actelion Pharmaceuticals Ltd., Sigilon Therapeutics Inc., BioMarin Pharmaceutical Inc., Merck & Co Inc., AstraZeneca PLC, Astellas Pharma Inc., Horizon Therapeutics plc, Leadient Biosciences Inc., Orphazyme A/S, Recordati Industria Chimica e Farmaceutica S.p.A., Valerion Therapeutics Llc, Viatris Inc., Chiese Farmaceutici SpA, Genzyme Corporation, Ultragenyx Pharmaceutical Inc., Sangamo Therapeutics Inc., Avrobio Inc., Axovant Gene Therapies Ltd., Regenxbio Inc., Krystal Biotech Inc., Homology Medicines Inc., Orchard Therapeutics plc, Sarepta Therapeutics Inc., Solid Biosciences Inc.

Which Regions Offer the Highest Growth Potential in the Lysosomal Disease Treatment Market?
North America was the largest region in the lysosomal disease treatment market in 2024.Europe is expected to be the fastest-growing region in the global lysosomal disease treatment market report during the forecast period. The regions covered in the lysosomal disease treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa

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