Lysosomal storage diseases (LSDs) are diseases caused by defects in single-genes. Enzyme defects cause nearly seventy percent of the LSDs, and the rest are defects in enzyme activator or associated proteins. A gene on a particular chromosome locus transcribes a particular enzyme, improper enzyme-coding results in inactive enzymes. Similarly, defective activators result from mutations in activator genes. These conditions cause disease of the organs in which they accumulate and decide the clinical signs and symptoms. Infants and children suffer more severely compared to adults. The clinical features are unique in many children and adults for the same disease. For example, the child's developing brain is more susceptible to insults and manifests symptoms and signs of dysfunction, while this may be milder or absent in adults. Enzyme testing is usually the initial diagnostic test, but genetic analysis of the gene mutations adds precision. There are many modern therapeutic techniques for these conditions. When applied early before organ damage sets in, these therapies have the potential to prevent or delay damage, improve quality of life, and increase lifespan.

Market Dynamics

Increasing significance of lysosomal storage diseases, arrangement of awareness programs by governments and the launch of new products are attributed to the growth of the global lysosomal storage diseases therapeutics market. For instance, on May 11, 2023, Chiesi USA, Inc., a U.S. based pharmaceutical company announced that its product Elfabrio was approved by U.S. Food and Drug Administration. It uses the enzyme pegunigalsidase alfa for enzyme replacement therapy (ERT) to treat adults with confirmed Fabry disease.

Key features of the study:

• This report provides an in-depth analysis of the global lysosomal storage diseases therapeutics market and provides market size (US$ Billion) and compound annual growth rate (CAGR) for the forecast period (2023–2030), considering 2022 as the base year
• It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market
• This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategies adopted by key players

• It profiles key players in the global lysosomal storage diseases therapeutics market based on the following parameters–Company highlights, products portfolio, key highlights, financial performance, and strategies
• Key companies covered as a part of this study include Shire plc, Pfizer, Inc., Sanofi, BioMarin Pharmaceutical Inc., Actelion Ltd., Raptor Pharmaceutical Corp., Protalix Biotherapeutics Inc., Quest Diagnostics, and Amicus Therapeutics, Inc.
• Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
• Global lysosomal storage diseases therapeutics market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
• Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global lysosomal storage diseases therapeutics market.

Detailed Segmentation:

• Global Lysosomal Storage Diseases Therapeutics Market, By Treatment:
  • Enzyme Replacement Therapy
  • Stem Cell Therapy
  • Substrate Reduction Therapy
  • Others
• Global Lysosomal Storage Diseases Therapeutics Market, By Indication:
  • Gaucher's Disease
  • Fabry Disease
  • Pompe’s Syndrome
  • Mucopolysaccharidosis
  • Others
• Global Lysosomal Storage Diseases Therapeutics Market, By End User:
  • Hospitals
  • Clinics
• Global Lysosomal Storage Diseases Therapeutics Market, By Geography:
  • North America
    
    • U.S.
    • Canada
  • Europe
    
    • U.K.
    • Germany
    • Italy
    • France
    • Spain
    • Russia
    • Rest of Europe
  • Asia Pacific
    
    • China
    • India
    • Japan
    • ASEAN
    • Australia
    • South Korea
    • Rest of Asia Pacific
  • Latin America
    
    • Brazil
    • Mexico
    • Argentina
    • Rest of Latin America
  • Middle East:
    
    • GCC
    • Israel
    • Rest of Middle East
  • Africa
    • Central Africa
    • South Africa
    • North Africa
• Company Profiles
  • Shire plc
  • Pfizer, Inc.
  • Sanofi SA
  • BioMarin Pharmaceutical Inc
  • Actelion Ltd.
  • Raptor Pharmaceutical Corp.
  • Protalix Biotherapeutics Inc.
  • Amicus Therapeutics, Inc.