Spinal Muscular Atrophy Market Positioned for Accelerated Development Through 2034, DelveInsight Finds | Novartis, Hoffmann-La Roche, Genentech, Inc., Scholar Rock, Roche, Cytokinetics, Biogen

The Key Spinal Muscular Atrophy Companies in the market include - Genentech, F. Hoffmann-La Roche, Scholar Rock, Biohaven/ Bristol-Myers Squibb, NMD Pharma, Novartis, Hoffmann-La Roche, Genentech, Inc., Scholar Rock, Roche, Cytokinetics, Biogen, Biohaven Pharmaceuticals, and others.

DelveInsiht's "Spinal Muscular Atrophy Market Insights, Epidemiology, and Market Forecast-2034 report offers an in-depth understanding of the Spinal Muscular Atrophy, historical and forecasted epidemiology as well as the Spinal Muscular Atrophy market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

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Some of the key facts of the Spinal Muscular Atrophy Market Report:

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The Spinal Muscular Atrophy market size is anticipated to grow with a significant CAGR during the study period (2020-2034)

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In June 2025, Biogen Inc. (Nasdaq: BIIB) has released topline results from its Phase 1 trial of salanersen (BIIB115/ION306), an antisense oligonucleotide (ASO) being developed for spinal muscular atrophy (SMA). Designed to offer greater potency than SPINRAZA (nusinersen) while using the same mechanism of action, salanersen aims to provide high efficacy with just one dose per year. Interim results from the study, which included SMA patients who had previously received gene therapy, were analyzed to support its progression to registrational trials. Both 40 mg and 80 mg annual doses were well-tolerated and showed significant slowing of neurodegeneration, with reductions in neurofilament levels and promising functional improvements, including the achievement of new WHO milestones.

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In March 2025, Novartis' experimental treatment OAV101 IT, an intrathecal version of the approved gene therapy onasemnogene abeparvovec (Zolgensma), has demonstrated promising results in improving patient performance on the Hammersmith Functional Motor Scale Expanded (HFMSE). These results, from the Phase 3 STEER clinical trial (NCT05089656), were recently presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.

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Among the three FDA-approved treatments, SPINRAZA and ZOLGENSMA are leading the market, with SPINRAZA securing a notable market share across the 7MM. These innovative therapies have revolutionized the treatment of Spinal Muscular Atrophy and delivered substantial financial gains for the pharmaceutical companies responsible for their creation.

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The classical forms of Spinal Muscular Atrophy are caused by biallelic mutations in the survival motor neuron 1 (SMN1) gene on chromosome 5, leading to a deficiency in SMN proteins. A second gene, SMN2, also generates SMN proteins, but they are typically truncated, with only 10%-20% remaining functional.

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Approximately 60% of newborns with Spinal Muscular Atrophy are diagnosed with type I, while types II and III account for the remaining 40%.

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Key Spinal Muscular Atrophy Companies: Genentech, F. Hoffmann-La Roche, Scholar Rock, Biohaven/ Bristol-Myers Squibb, NMD Pharma, Novartis, Hoffmann-La Roche, Genentech, Inc., Scholar Rock, Roche, Cytokinetics, Biogen, Biohaven Pharmaceuticals, and others

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Key Spinal Muscular Atrophy Therapies: ZOLGENSMA (onasemnogene abeparvovac-xioi), EVRYSDI (risdiplam), SPINRAZA, Apitegromab (SRK-015), Taldefgrobep Alfa (BHV-2000), GYM329/RG6237, NMD670, branaplam, RO7204239, Risdiplam, Apitegromab, GYM329, Reldesemtiv, Nusinersen, taldefgrobep alfa, and others

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The Spinal Muscular Atrophy epidemiology based on gender analyzed that it can be concluded that Spinal Muscular Atrophy (SMA) occurs equally in men and women

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The Spinal Muscular Atrophy market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Spinal Muscular Atrophy pipeline products will significantly revolutionize the Spinal Muscular Atrophy market dynamics.

Spinal Muscular Atrophy Overview

Spinal muscular atrophy (SMA) is an autosomal recessive rare neuromuscular disorder that is characterized by progressive muscle wasting because of the loss of motor neurons in the spinal cord (anterior horn), which controls voluntary muscle movement. Spinal Muscular Atrophy is one of the most common causes of infant death and mobility impairment. It mostly affects proximal muscles and respiratory muscles. The most common form of SMA is known as '5q SMA' due to its genetic cause

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Spinal Muscular Atrophy Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Spinal Muscular Atrophy Epidemiology Segmentation:

The Spinal Muscular Atrophy market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:

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Total Prevalence of Spinal Muscular Atrophy

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Prevalent Cases of Spinal Muscular Atrophy by severity

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Gender-specific Prevalence of Spinal Muscular Atrophy

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Diagnosed Cases of Episodic and Chronic Spinal Muscular Atrophy

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Spinal Muscular Atrophy Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Spinal Muscular Atrophy market or expected to get launched during the study period. The analysis covers Spinal Muscular Atrophy market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Spinal Muscular Atrophy Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Spinal Muscular Atrophy Therapies and Key Companies

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ZOLGENSMA (onasemnogene abeparvovac-xioi): Novartis

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EVRYSDI (risdiplam): Genentech/ F. Hoffmann-La Roche

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SPINRAZA: Biogen

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Apitegromab (SRK-015): Scholar Rock

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Taldefgrobep Alfa (BHV-2000): Biohaven/ Bristol-Myers Squibb

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GYM329/RG6237: Roche

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NMD670: NMD Pharma

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branaplam: Novartis

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RO7204239: Hoffmann-La Roche

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Risdiplam: Genentech, Inc.

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Apitegromab: Scholar Rock

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GYM329: Roche

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Reldesemtiv: Cytokinetics

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Nusinersen: Biogen

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taldefgrobep alfa: Biohaven Pharmaceuticals

Discover more about therapies set to grab major Spinal Muscular Atrophy market share @ Spinal Muscular Atrophy Treatment Market [https://www.delveinsight.com/sample-request/spinal-muscular-atrophy-market-insights-epidemiology-and-market-forecast?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Spinal Muscular Atrophy Market Drivers

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Newborn Screening for Spinal Muscular Atrophy

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Promising Upcoming Launches and Approval

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Raising Awareness for Spinal Muscular Atrophy

Spinal Muscular Atrophy Market Barriers

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High Spinal Muscular Atrophy treatment Cost

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Competition for Spinal Muscular Atrophy Emerging Therapies

Scope of the Spinal Muscular Atrophy Market Report

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Study Period: 2020-2034

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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Key Spinal Muscular Atrophy Companies: Genentech, F. Hoffmann-La Roche, Scholar Rock, Biohaven/ Bristol-Myers Squibb, NMD Pharma, Novartis, Hoffmann-La Roche, Genentech, Inc., Scholar Rock, Roche, Cytokinetics, Biogen, Biohaven Pharmaceuticals, and others

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Key Spinal Muscular Atrophy Therapies: ZOLGENSMA (onasemnogene abeparvovac-xioi), EVRYSDI (risdiplam), SPINRAZA, Apitegromab (SRK-015), Taldefgrobep Alfa (BHV-2000), GYM329/RG6237, NMD670, branaplam, RO7204239, Risdiplam, Apitegromab, GYM329, Reldesemtiv, Nusinersen, taldefgrobep alfa, and others

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Spinal Muscular Atrophy Therapeutic Assessment: Spinal Muscular Atrophy current marketed and Spinal Muscular Atrophy emerging therapies

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Spinal Muscular Atrophy Market Dynamics: Spinal Muscular Atrophy market drivers and Spinal Muscular Atrophy market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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Spinal Muscular Atrophy Unmet Needs, KOL's views, Analyst's views, Spinal Muscular Atrophy Market Access and Reimbursement

To know more about Spinal Muscular Atrophy companies working in the treatment market, visit @ Spinal Muscular Atrophy Clinical Trials and Therapeutic Assessment [https://www.delveinsight.com/sample-request/spinal-muscular-atrophy-market-insights-epidemiology-and-market-forecast?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Table of Contents

1. Spinal Muscular Atrophy Market Report Introduction

2. Executive Summary for Spinal Muscular Atrophy

3. SWOT analysis of Spinal Muscular Atrophy

4. Spinal Muscular Atrophy Patient Share (%) Overview at a Glance

5. Spinal Muscular Atrophy Market Overview at a Glance

6. Spinal Muscular Atrophy Disease Background and Overview

7. Spinal Muscular Atrophy Epidemiology and Patient Population

8. Country-Specific Patient Population of Spinal Muscular Atrophy

9. Spinal Muscular Atrophy Current Treatment and Medical Practices

10. Spinal Muscular Atrophy Unmet Needs

11. Spinal Muscular Atrophy Emerging Therapies

12. Spinal Muscular Atrophy Market Outlook

13. Country-Wise Spinal Muscular Atrophy Market Analysis (2020-2034)

14. Spinal Muscular Atrophy Market Access and Reimbursement of Therapies

15. Spinal Muscular Atrophy Market Drivers

16. Spinal Muscular Atrophy Market Barriers

17. Spinal Muscular Atrophy Appendix

18. Spinal Muscular Atrophy Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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