US Sanfilippo Syndrome (MPS-III) Therapeutics Market Report 2023 to 2030

US Sanfilippo Syndrome (MPS-III) Therapeutics Market Analysis

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US Sanfilippo Syndrome (MPS-III) Therapeutics market is projected to grow from $xx Mn in 2023 to $xx Mn by 2030, registering a CAGR of xx% during the forecast period of 2023 - 2030. The market for Sanfilippo Syndrome (MPS-III) Therapeutics is growing rapidly as a result of increase in research and development activities, patient support and advocacy programmes, launch of new emerging therapies, increase in healthcare spending globally. Phoenix Nest Inc, Allievex Corp, Amicus Therapeutics Inc, ArmaGen Inc, Esteve Pharmaceuticals SA, JCR Pharmaceuticals Co Ltd, M6P Therapeutics, Orchard Therapeutics Plc, Seelos Therapeutics, Inc., and BioStrategies LC are top key players operating in global Sanfilippo Syndrome (MPS-III) Therapeutics market.

ID: IN10USRD065 CATEGORY: Rare Diseases GEOGRAPHY: US AUTHOR: Nipun Arora

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US Sanfilippo Syndrome (MPS-III) Therapeutics Market Analysis Summary

US Sanfilippo Syndrome (MPS-III) Therapeutics Market is valued at around $0.47 Bn in 2022 and is projected to reach $0.57 Bn by 2030, exhibiting a CAGR of 2.6% during the forecast period 2023-2030.

Mucopolysaccharidosis type III (MPS III) also called Sanfilippo syndrome is a hereditary metabolic disorder brought on by a lack of or an issue with specific enzymes required to degrade compounds called glycosaminoglycans (GAG). It is a neurodegenerative disease with seizures, hyperactivity, liver and spleen enlargement, severe diarrhoea or constipation, and tonsils and adenoid enlargement as symptoms. Sanfilippo syndrome has no known, accepted treatment. However, there are various (some conventional, some unique) treatment options for Sanfilippo syndrome, including hematopoietic stem cell transplantation, enzyme replacement therapy, the use of pharmacological chaperones, and gene therapy. The effectiveness of hematopoietic stem cell transplantation, which involves the transplants, was greatly questioned due to its potential to provide neurological advantages and safety. Enzyme replacement therapy (ERT) clinical studies are now being conducted to treat Sanfilippo syndrome MPS IIIA (intrathecal) and MPS IIIB (intravenous). Furthermore, the same will begin for Gene therapy. The most practical method for correcting genes is gene therapy, despite its extreme complexity. Another difficulty with the treatment is the need to implant the healthy gene into hundreds of millions of cells that already have the faulty gene. The market size for Sanfilippo syndrome will further increase with the introduction of new treatments for the condition, including SLS-005 (trehalose), Lysogene (LYS-SAF302), Tralesinidase alfa (BMN 250), and others. Amicus Therapeutics Inc, ArmaGen Inc, Esteve Pharmaceuticals SA, JCR Pharmaceuticals Co Ltd, M6P Therapeutics, Orchard Therapeutics Plc, Seelos Therapeutics, Inc., and BioStrategies LC are top key players operating in global Sanfilippo Syndrome (MPS-III) Therapeutics market.

Market Dynamics

Market Drivers

Improvement in diagnostics methodologies, increase in understanding of the disease, increase in healthcare spending across the world, increase in research and development activities, patient support and advocacy programmes and launch of new therapies like Enzyme replacement therapy, gene therapy CRISPR-Cas9 and substrate reduction therapy all act as a market growth driver.

The launch of emerging therapies such as SLS-005 (trehalose), Lysogene (LYS-SAF302), Tralesinidase alfa (BMN 250) and others will add to the Sanfilippo syndrome market size.

Market Restraints

The difficulty of the administered drugs in crossing the blood-brain barrier and the death of neurons in MPS-III acts as a restraint. Limited patient populations, difficulties in diagnosis, and high costs associated with research and development also act as market growth restraints.

Key players

Sangamo Therapeutics, Inc. REGENXBIO Inc. Abeona Therapeutics Inc. Lysogene Esteve Pharmaceuticals Orchard Therapeutics plc Denali Therapeutics Inc. Ultragenyx Pharmaceutical Inc. Shire (AbbVie Inc.) Phoenix Nest, Inc.

1. Executive Summary
1.1 Disease Overview
1.2 Global Scenario
1.3 Country Overview
1.4 Healthcare Scenario in Country
1.5 Patient Journey
1.6 Health Insurance Coverage in Country
1.7 Active Pharmaceutical Ingredient (API)
1.8 Recent Developments in the Country

2. Market Size and Forecasting
2.1 Epidemiology of Disease
2.2 Market Size (With Excel & Methodology)
2.3 Market Segmentation (Check all Segments in Segmentation Section)

3. Market Dynamics
3.1 Market Drivers
3.2 Market Restraints

4. Competitive Landscape
4.1 Major Market Share

4.2 Key Company Profile (Check all Companies in the Summary Section)

4.2.1 Company
4.2.1.1 Overview
4.2.1.2 Product Applications and Services
4.2.1.3 Recent Developments
4.2.1.4 Partnerships Ecosystem
4.2.1.5 Financials (Based on Availability)

5. Reimbursement Scenario
5.1 Reimbursement Regulation
5.2 Reimbursement Process for Diagnosis
5.3 Reimbursement Process for Treatment

6. Methodology and Scope

Market Segmentations For US Sanfilippo Syndrome (MPS-III) Therapeutics market

By Target

Alpha N-Acetylglucosaminidase
N Sulphoglucosamine Sulphohydrolase
Polyadenylate Binding Protein 2
Heparan Alpha Glucosaminide N-Acetyltransferase
Interleukin 1 Receptor Type 1
N-Acetylgalactosamine 6 Sulfatase

By Treatment type

Gene therapy
Recombinant enzyme
Fusion protein
Gene-modified cell therapy
Small Molecule
Enzyme

By Route of administration

Intravenous
Intrathecal
Intracerebral
Nasal
Oral

Methodology for Database Creation

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Analyzing historical proprietary data collected from multiple projects.
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Combining Secondary and Primary Research

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