The global spinal muscular atrophy treatment market size was valued at USD 4.6 billion in 2023, driven by the high prevalence of the disorder, spreading awareness, and technological advancements in diagnostics and treatment. The market size is anticipated to grow at a CAGR of 23.2% during the forecast period of 2024-2032 to achieve a value of USD 29.9 billion by 2032.
Spinal muscular atrophy is an genetic disorder which weakens skeletal muscles, resulting in them wasting away. It is caused by gene defects that result in loss of motor neurons involved in controlling muscle movement. No cure has yet been developed for spinal muscular atrophy, but treatments include managing the symptoms and prevention of further complications.
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Based on disease type, the market can be divided into Type 1 SMA, Type 2 SMA, Type 3 SMA, and Type 4 SMA. By treatment, the market is bifurcated into gene replacement therapy and drug therapy. On the basis of route of administration, the market is segmented into oral and intrathecal. Based on end use, the market is categorised into hospitals, homecare, and specialty clinics, among others.
On the basis of distribution channel, the market is segmented into hospital pharmacies and retail pharmacies, among others. By region, the market is divided into North America, Europe, Asia Pacific, Latin America, and Middle East and Africa.
The comprehensive EMR report provides an in-depth assessment of the market based on the Porter's five forces model along with giving a SWOT analysis. The report gives a detailed analysis of the following key players in the global spinal muscular atrophy treatment market covering their competitive landscape and latest developments like mergers, acquisitions, investments, and expansion plans.
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Type 1 SMA has accounted for a larger share of the spinal muscular atrophy treatment market among other disease types and is expected to do the same in the forecast period. This is primarily because maximum cases of spinal muscular atrophy disorder are of Type 1.
Also known as Werdnig-Hoffmann disease, spinal musical atrophy Type 1 affects infants with symptoms becoming visible before the age of 6 months. Without intervention and treatment, Type 1 SMA can lead to fatalities before the age of 2 years. Due to the wide prevalence of type 1 SMA and timely diagnosis, treatments for Type 1 occupy a leading share of the market.
Type 2 SMA is the second most common occurring form of spinal musical atrophy, occurring between the ages of 6 months and 18 months, with progressively worsening symptoms. Spinal muscular atrophy treatment helps extend the lifespan and improve quality of life for patients with Type 2, increasing the demand for the market for SMA treatment.
Hospital pharmacies has been leading the spinal muscular atrophy treatment market on the basis of distribution channels and are expected to be leading the market in the forecast period as well. Medications developed for the treatment of the spinal muscular atrophy are primarily administered intravenously and are advised to be administered by professional doctors or medical experts only. These factors are largely and rapidly contributing to the growth of the hospital pharmacy segment but are acting as a major restraining factor for the growth of the retail pharmacy segment.
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Biogen Inc is a biotechnology company that develops therapy for the treatment of neurological diseases globally with the help of world’s top neuroscientists and neurologists. It works with an aim to improve the future of medical research. It has its headquarter in Massachusetts, United States and was founded in 1978.
Ionis Pharmaceuticals Inc. was established in 1989 and is based out of California in the United States. It is a biotechnology company that is involved in the development and discovery of RNA- targeted therapeutics. They have a drug development platform named antisense technology that is capable of treating illnesses where other treatments prove to be ineffective.
F. Hoffmann-La Roche Ltd. was founded in 1896 with headquarters in Basel, Switzerland. It is a healthcare company that provides its products globally. They develop drugs that are used for the treatment of metabolic diseases, cancer, and virus diseases.
Other market players include Novartis AG, and Cytokinetics Inc, among others.
|Scope of the Report||
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
|Breakup by Treatment Type||
|Breakup by Diseases Type||
|Breakup by Population Type||
|Breakup by Dosage Form||
|Breakup by Route of Administration||
|Breakup by End User||
|Breakup by Treatment Channel||
|Breakup by Distribution Channel||
|Breakup by Region||
*At Expert Market Research, we strive to always give you current and accurate information. The numbers depicted in the description are indicative and may differ from the actual numbers in the final EMR report.
1.1 Objectives of the Study
1.2 Key Assumptions
1.3 Report Coverage – Key Segmentation and Scope
1.4 Research Methodology
2 Executive Summary
3 Spinal Muscular Atrophy Overview
3.1 Guidelines and Stages
3.3 Screening and Diagnosis
3.4 Treatment Pathway
4 Patient Profile
4.1 Patient Profile Overview
4.2 Patient Psychology and Emotional Impact Factors
4.3 Risk Assessment and Treatment Success Rate
5 Spinal Muscular Atrophy Epidemiology Analysis
5.1 Epidemiology Overview (2016-2031)
5.2 North America Spinal Muscular Atrophy Epidemiology (2016-2031)
5.3 Europe Spinal Muscular Atrophy Epidemiology (2016-2031)
5.4 Asia-Pacific Spinal Muscular Atrophy Epidemiology (2016-2031)
5.5 Latin America Spinal Muscular Atrophy Epidemiology (2016-2031)
5.6 Middle East & Africa Spinal Muscular Atrophy Epidemiology (2016-2031)
6 Global Spinal Muscular Atrophy Treatment Market Overview
6.1 Global Spinal Muscular Atrophy Treatment Market Historical Value (2017-2023)
6.2 Global Spinal Muscular Atrophy Treatment Market Forecast Value (2024-2032)
7 Global Spinal Muscular Atrophy Treatment Market Landscape
7.1 Global Spinal Muscular Atrophy Treatment: Developers Landscape
7.1.1 Analysis by Year of Establishment
7.1.2 Analysis by Company Size
7.1.3 Analysis by Region
7.2 Global Spinal Muscular Atrophy Treatment Product Landscape
7.2.1 Analysis by Treatment Type
7.2.2 Analysis by Disease Type
7.2.3 Analysis by End User
8 Spinal Muscular Atrophy Treatment Challenges and Unmet Needs
8.1 Treatment Pathway Challenges
8.2 Compliance and Drop-Out Analysis
8.3 Awareness and Prevention Gaps
9 Cost of Treatment
10 Global Spinal Muscular Atrophy Treatment Market Dynamics
10.1 Market Drivers and Constraints
10.2 SWOT Analysis
10.3 Porter’s Five Forces Model
10.4 Key Demand Indicators
10.5 Key Price Indicators
10.6 Industry Events, Initiatives, and Trends
10.7 Value Chain Analysis
11 Global Spinal Muscular Atrophy Treatment Market Segmentation
11.1 Global Spinal Muscular Atrophy Treatment Market by Treatment Type
11.1.1 Market Overview
11.1.2 Gene Modifying Drugs
11.1.3 Gene Replacement Therapy
11.1.4 Physical Therapy
11.2 Global Spinal Muscular Atrophy Treatment Market by Diseases Type
11.2.1 Market Overview
11.3 Global Spinal Muscular Atrophy Treatment Market by Population Type
11.3.1 Market Overview
11.4 Global Spinal Muscular Atrophy Treatment Market by Dosage Form
11.4.1 Market Overview
11.5 Global Spinal Muscular Atrophy Treatment Market by Route of Administration
11.5.1 Market Overview
11.6 Global Spinal Muscular Atrophy Treatment Market by End User
11.6.1 Market Overview
11.6.3 Specialty Clinics
11.7 Global Spinal Muscular Atrophy Treatment Market by Treatment Channel
11.7.1 Market Overview
11.8 Global Spinal Muscular Atrophy Treatment Market by Distribution Channel
11.8.1 Market Overview
11.8.2 Hospital Pharmacy
11.8.3 Online Pharmacy
11.8.4 Retail Pharmacy
11.9 Global Spinal Muscular Atrophy Treatment Market by Region
11.9.1 Market Overview
11.9.2 North America
11.9.4 Asia Pacific
11.9.5 Latin America
11.9.6 Middle East and Africa
12 North America Spinal Muscular Atrophy Treatment Market
12.1 Market Share by Country
12.2 United States of America
13 Europe Spinal Muscular Atrophy Treatment Market
13.1 Market Share by Country
13.2 United Kingdom
14 Asia Pacific Spinal Muscular Atrophy Treatment Market
14.1 Market Share by Country
15 Latin America Spinal Muscular Atrophy Treatment Market
15.1 Market Share by Country
16 Middle East and Africa Spinal Muscular Atrophy Treatment Market
16.1 Market Share by Country
16.2 Saudi Arabia
16.3 United Arab Emirates
16.5 South Africa
17 Regulatory Framework
17.1 Regulatory Overview
17.1.1 US FDA
17.1.2 EU EMA
17.1.3 INDIA CDSCO
17.1.4 JAPAN PMDA
18 Patent Analysis
18.1 Analysis by Type of Patent
18.2 Analysis by Publication year
18.3 Analysis by Issuing Authority
18.4 Analysis by Patent Age
18.5 Analysis by CPC Analysis
18.6 Analysis by Patent Valuation
18.7 Analysis by Key Players
19 Grants Analysis
19.1 Analysis by year
19.2 Analysis by Amount Awarded
19.3 Analysis by Issuing Authority
19.4 Analysis by Grant Application
19.5 Analysis by Funding Institute
19.6 Analysis by NIH Departments
19.7 Analysis by Recipient Organization
20 Clinical Trials Analysis
20.1 Analysis by Trial Registration Year
20.2 Analysis by Trial Status
20.3 Analysis by Trial Phase
20.4 Analysis by Therapeutic Area
20.5 Analysis by Geography
21 Funding and Investment Analysis
21.1 Analysis by Funding Instances
21.2 Analysis by Type of Funding
21.3 Analysis by Funding Amount
21.4 Analysis by Leading Players
21.5 Analysis by Leading Investors
21.6 Analysis by Geography
22 Partnership and Collaborations Analysis
22.1 Analysis by Partnership Instances
22.2 Analysis by Type of Partnership
22.3 Analysis by Leading Players
22.4 Analysis by Geography
23 Supplier Landscape
23.1.1 Financial Analysis
23.1.2 Product Portfolio
23.1.3 Demographic Reach and Achievements
23.1.4 Mergers and Acquisitions
23.2 F. Hoffmann-La Roche Ltd.
23.2.1 Financial Analysis
23.2.2 Product Portfolio
23.2.3 Demographic Reach and Achievements
23.2.4 Mergers and Acquisitions
23.3 Boehringer Ingelheim International GmbH.
23.3.1 Financial Analysis
23.3.2 Product Portfolio
23.3.3 Demographic Reach and Achievements
23.3.4 Mergers and Acquisitions
23.4 GSK plc.
23.4.1 Financial Analysis
23.4.2 Product Portfolio
23.4.3 Demographic Reach and Achievements
23.4.4 Mergers and Acquisitions
23.5 Novartis AG
23.5.1 Financial Analysis
23.5.2 Product Portfolio
23.5.3 Demographic Reach and Achievements
23.5.4 Mergers and Acquisitions
23.6.1 Financial Analysis
23.6.2 Product Portfolio
23.6.3 Demographic Reach and Achievements
23.6.4 Mergers and Acquisitions
23.7 Pfizer Inc.
23.7.1 Financial Analysis
23.7.2 Product Portfolio
23.7.3 Demographic Reach and Achievements
23.7.4 Mergers and Acquisitions
23.8 Ionis Pharmaceuticals, Inc.
23.8.1 Financial Analysis
23.8.2 Product Portfolio
23.8.3 Demographic Reach and Achievements
23.8.4 Mergers and Acquisitions
23.9 Regeneron Pharmaceuticals Inc.
23.9.1 Financial Analysis
23.9.2 Product Portfolio
23.9.3 Demographic Reach and Achievements
23.9.4 Mergers and Acquisitions
23.10.1 Financial Analysis
23.10.2 Product Portfolio
23.10.3 Demographic Reach and Achievements
23.10.4 Mergers and Acquisitions
23.11 AbbVie Inc.
23.11.1 Financial Analysis
23.11.2 Product Portfolio
23.11.3 Demographic Reach and Achievements
23.11.4 Mergers and Acquisitions
23.12 Novo Nordisk
23.12.1 Financial Analysis
23.12.2 Product Portfolio
23.12.3 Demographic Reach and Achievements
23.12.4 Mergers and Acquisitions
23.13 Takeda Pharmaceutical Company Limited
23.13.1 Financial Analysis
23.13.2 Product Portfolio
23.13.3 Demographic Reach and Achievements
23.13.4 Mergers and Acquisitions
23.14.1 Financial Analysis
23.14.2 Product Portfolio
23.14.3 Demographic Reach and Achievements
23.14.4 Mergers and Acquisitions
23.15 Leadiant Biosciences, Inc.
23.15.1 Financial Analysis
23.15.2 Product Portfolio
23.15.3 Demographic Reach and Achievements
23.15.4 Mergers and Acquisitions
23.16 Catalyst Pharmaceuticals, Inc.
23.16.1 Financial Analysis
23.16.2 Product Portfolio
23.16.3 Demographic Reach and Achievements
23.16.4 Mergers and Acquisitions
23.17 PTC Therapeutics
23.17.1 Financial Analysis
23.17.2 Product Portfolio
23.17.3 Demographic Reach and Achievements
23.17.4 Mergers and Acquisitions
24 Global Spinal Muscular Atrophy Treatment Market- Distribution Model (Additional Insight)
24.2 Potential Distributors
24.3 Key Parameters for Distribution Partner Assessment
25 Key Opinion Leaders (KOL) Insights (Additional Insight)
26 Company Competitiveness Analysis (Additional Insight)
26.1 Very Small Companies
26.2 Small Companies
26.3 Mid-Sized Companies
26.4 Large Companies
26.5 Very Large Companies
27 Payment Methods (Additional Insight)
27.1 Government Funded
27.2 Private Insurance
*Additional insights provided are customisable as per client requirements.
In 2023, the global market for spinal muscular atrophy treatment attained a value of USD 4.6 billion.
The market is anticipated to grow at a CAGR of 23.2% during the forecast period of 2024-2032 to reach a value of USD 29.9 billion by 2032.
The major market drivers include prevalence of the disorder, spreading awareness, and technological advancements in diagnostics and treatment.
The key trends of the market are new product launches, increased research and development, prenatal testing, and adoption of expensive drugs.
Key market players include Biogen Inc., Ionis Pharmaceuticals Inc., F. Hoffmann-La Roche Ltd, Novartis AG, and Cytokinetics Inc, among others.
Spinal muscular atrophy is a genetic disorder in which skeletal muscles are weakened and waste away.
It is caused by gene defects that result in loss of motor neurons involved in controlling muscle movement.
The global spinal muscular atrophy treatment market is segmented based on disease type, treatment, route of administration, end use, distribution channel, and region.
No cure has yet been developed for spinal muscular atrophy, but treatments include drug therapy and gene replacement therapy that are aimed at managing the symptoms and preventing complications.
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