Lysosomal Storage Diseases Therapeutics Market, By Treatment (Enzyme Replacement Therapy (Stem Cell Therapy, Substrate Reduction Therapy, Others)), By Indication (Gaucher's Disease, Fabry Disease, Pompe’s Syndrome, Mucopolysaccharidosis, Others), By End U

Lysosomal Storage Diseases Therapeutics Market, By Treatment (Enzyme Replacement Therapy (Stem Cell Therapy, Substrate Reduction Therapy, Others)), By Indication (Gaucher's Disease, Fabry Disease, Pompe’s Syndrome, Mucopolysaccharidosis, Others), By End User (Hospitals, Clinics), and By Region (North America Latin America Europe Asia Pacific Middle East Africa) - Size, Share, Outlook, and Opportunity Analysis, 2022 - 2028

The deficiency of enzymes that causes a specific disease condition called lysosomal storage disease includes diseases such as Fabry Disease, Gaucher disease, lysosomal acid lipase deficiency, mucopolysaccharidosis, Hunter syndrome, and Pompe disease. Enzyme replacement therapy involves intravenous administration of enzymes in order to correct the deficiency of enzymes. Enzyme replacement is associated with fewer side effects than other treatment methods. Enzymes are obtained from sources such as human cells, animal cells, and recombinant DNA technology. Enzyme replacement, although does not provide permanent cure, helps prevents permanent damage to the body caused due to deficiency of a specific enzyme. Treatment consists of weekly or monthly doses depending upon the disease.

Restraints of the Global Lysosomal Storage Diseases Therapeutics Market

Factors such as high cost associated with the enzyme replacement therapy and complex reimbursement scenario causing therapy to be excluded from coverage by insurers due to this being rare diseases, hamper growth of the market over the forecast period.

Key features of the study:

This report provides in-depth analysis of the global lysosomal storage diseases therapeutics market and provides market size (US$ Million) and compound annual growth rate (CAGR %) for the forecast period (2022-2028), considering 2021, as the base year
It elucidates potential revenue opportunity across different segments and explains attractive investment proposition matrix for this market
This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, regional outlook, and competitive strategy adopted by leading players
It profiles leading players in the global lysosomal storage diseases therapeutics market based on the following parameters – company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments, strategies, and future plans
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 companies to make informed decision regarding future product launches, technology up-gradation, market expansion, and marketing tactics
The 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 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
By Treatment
Enzyme Replacement Therapy
Stem Cell Therapy
Substrate Reduction Therapy
Others
By Indication
Gaucher's Diseases
Fabry Diseases
Pompe’s Syndrome
Mucopolysaccharidosis
Other
By End User
Hospitals
Clinics
By Country:
U.S.
Canada
Europe
By Treatment
Enzyme Replacement Therapy
Stem Cell Therapy
Substrate Reduction Therapy
Others
By Indication
Gaucher's Diseases
Fabry Diseases
Pompe’s Syndrome
Mucopolysaccharidosis
Other
By End User
Hospitals
Clinics
By Country:
U.K.
Germany
Italy
France
Spain
Russia
Rest of Europe
Asia Pacific
By Treatment
Enzyme Replacement Therapy
Stem Cell Therapy
Substrate Reduction Therapy
Others
By Indication
Gaucher's Diseases
Fabry Diseases
Pompe’s Syndrome
Mucopolysaccharidosis
Other
By End User
Hospitals
Clinics
By Country:
China
India
Japan
ASEAN
Australia
South Korea
Rest of Asia Pacific
Latin America
By Treatment
Enzyme Replacement Therapy
Stem Cell Therapy
Substrate Reduction Therapy
Others
By Indication
Gaucher's Diseases
Fabry Diseases
Pompe’s Syndrome
Mucopolysaccharidosis
Other
By End User
Hospitals
Clinics
By Country:
Brazil
Mexico
Argentina
Rest of Latin America
Middle East:
By Treatment
Enzyme Replacement Therapy
Stem Cell Therapy
Substrate Reduction Therapy
Others
By Indication
Gaucher's Diseases
Fabry Diseases
Pompe’s Syndrome
Mucopolysaccharidosis
Other
By End User
Hospitals
Clinics
By Country:
GCC
Israel
Rest of Middle East
Africa
By Treatment
Enzyme Replacement Therapy
Stem Cell Therapy
Substrate Reduction Therapy
Others
By Indication
Gaucher's Diseases
Fabry Diseases
Pompe’s Syndrome
Mucopolysaccharidosis
Other
By End User
Hospitals
Clinics
By Country/Region:
Central Africa
South Africa
North Africa
Company Profiles
Shire plc*
Company Overview
Product Portfolio
Financial Performance
Key Strategies
Recent Developments
Pfizer, Inc.
Sanofi SA
BioMarin Pharmaceutical Inc
Actelion Ltd.
Raptor Pharmaceutical Corp.
Protalix Biotherapeutics Inc.
Amicus Therapeutics, Inc.

“*” marked represents similar segmentation in other categories in the respective section.