Spinal Muscular Atrophy Treatment Report 2025 – For Strategy Officers And Market Intelligence Teams

The Business Research Company's Spinal Muscular Atrophy Treatment Report 2025 – For Strategy Officers And Market Intelligence Teams

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How Big Is The Global Spinal Muscular Atrophy Treatment Market Expected To Grow?

The spinal muscular atrophy treatment market size has seen substantial growth in recent years, expanding from $4.41 billion in 2024 to expected $5.1 billion in 2025 at a compound annual growth rate CAGR of 15.7%. This growth has been mainly influenced by factors such as increasing prevalence of spinal muscular atrophy, rising approvals of novel gene therapies, growing awareness of early diagnosis and treatment, increasing investment in research and development, and the rising availability of newborn screening programs.

What Is Its Annual Growth Rate?

The forecast also indicates that the spinal muscular atrophy treatment market will continue evolving at a fast pace. It is anticipated to escalate to $9.02 billion by 2029 at a compound annual growth rate CAGR of 15.3%.

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What Is Driving The Growth Of The Spinal Muscular Atrophy Treatment Market?

In the forecast period, the contributions to the market's growth will mirror those of the historic period still, focusing on gene therapy techniques, personalized medicine approaches, technology-driven oral therapies, developments in newborn screening programs, and combination therapy strategies. One key driver of this growth is the burgeoning focus on gene therapy, a medical technique that modifies an individual's genetic material, i.e., DNA or RNA, to treat or prevent diseases.
Gene therapy is highly regarded because of its potential to essentially treat or cure the origin of genetic disorders by directly modifying or replacing faulty genes. This results in permanent therapeutic effects which override those of traditional treatments. For instance, onasemnogene abeparvovec Zolgensma is a spinal muscular atrophy treatment useful for gene therapy. It provides a one-time, targeted approach that delivers a functional copy of the SMN1 gene, which significantly improves motor function and quality of life for patients with SMA.
In January 2024, according to the American Society of Gene and Cell Therapy, the number of gene therapies in Phase III trials increased by 10% in the fourth quarter of 2023, marking the first such increase since the third quarter of 2022.

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Which Leading Companies Are Driving The Growth Of The Spinal Muscular Atrophy Treatment Market Share?
Key industry players operating in the spinal muscular atrophy treatment market are F. Hoffmann-La Roche Ltd, Novartis AG, Biogen Inc., Chugai Pharmaceutical Co. Ltd, PTC Therapeutics Inc., and others. They are leading the growth in the sector with their innovative solutions and strategies.

What Are The Key Trends Of The Spinal Muscular Atrophy Treatment Market?
An emerging trend shows that these companies are developing innovative formulations such as survival motor neuron 2 SMN2 splicing modifier tablets to enhance motor function and slow disease progression. For instance, Genentech Inc., a US-based biotechnology company, received U.S. Food and Drug Administration approval for its Evrysdi risdiplam tablet in February 2025. This tablet is the first and only non-invasive SMN2 splicing modifier tablet for the treatment of SMA on the market, offering more convenience and flexibility in disease management.

How Is The Global Spinal Muscular Atrophy Treatment Market Segmented?

The spinal muscular atrophy treatment market includes various segments:

1 By Treatment: Medication, Physical Therapy, Surgery, Other Treatments
2 By Type: Werdnig-Hoffmann Disease, Infant Spinal Muscular Atrophy, Kugelberg-Welander Disease, Adult Spinal Muscular Atrophy
3 By Route Of Administration: Oral, Parenteral
4 By Distribution Channel: Hospital Pharmacies, Retail Pharmacies, Other Distribution Channels
5 By End User: Hospitals, Homecare, Specialty Clinics, Other End Users

What Are The Leading Region In The Spinal Muscular Atrophy Treatment Market?

North America was the largest region in the spinal muscular atrophy treatment market in 2024. However, Asia-Pacific is expected to be the fastest-growing region in the coming years.

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