Emerging Spinal Muscular Atrophy Market Driver 2025-2034: Impact Of Rising Prevalence Of Rare Genetic Diseases On the Market

How Is the Spinal Muscular Atrophy Market Projected to Grow, and What Is Its Market Size?
The market size for spinal muscular atrophy has seen a swift rise in recent years. There will be a jump from $3.53 billion in 2024 to $4.01 billion in 2025, following a compound annual growth rate (CAGR) of 13.5%. Several factors have contributed to the growth in this period, including the advancement in diagnosis and early detection, escalating instances of rare genetic diseases, enhancements in healthcare infrastructure, support from government and regulatory bodies, as well as the surge in unmet healthcare requirements.

The market size for spinal muscular atrophy is predicted to experience quick expansion in the coming years. The expected growth is to a staggering $6.59 billion by 2029, at a compound annual growth rate (CAGR) of 13.2%. This projected increase in the forecast period is chiefly due to factors such as heightened awareness and diagnosis, wider access to treatments, enhanced health care investments in rare diseases, a supportive regulatory framework, and increased healthcare expenditure. The forecast period is also expected to see trends like advancements in oral therapies, the embrace of gene therapy, technological progress in drug delivery systems, the utilisation of artificial intelligence in the development of SMA treatments, and gene-editing technologies.

What Are the Key Drivers Behind the Growth of the Spinal Muscular Atrophy Market?
The growth of the spinal muscular atrophy market is predicted to be fueled by the increasing occurrence of rare genetic diseases. Such diseases, which derive from gene mutations and affect a small fraction of the population, often lead to serious, long-term health issues. The surge in rare genetic diseases can be credited to enhancements in diagnostic technologies, heightened awareness, and improved access to genetic testing, all of which contribute to a higher number of cases being identified and reported. One such rare genetic condition is spinal muscular atrophy (SMA), which presents both challenges and possibilities in the exploration of inherited diseases, underscoring the necessity for continued genetic research. For example, a report published by the UK-based Office for National Statistics in July 2023 stated that genetic testing of SMN1 has facilitated exact epidemiological studies. These studies have disclosed that SMA is seen in 1 out of 10,000 to 20,000 live births and that over 95% of the patients afflicted carry a homozygous deletion for SMN1. Hence, the increasing occurrence of rare genetic illnesses is set to stimulate the expansion of the spinal muscular atrophy market.

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Who Are the Major Industry Players Fueling Spinal Muscular Atrophy Market Expansion?
Major companies operating in the spinal muscular atrophy market are Pfizer Inc., F. Hoffmann-La Roche Ltd, Bayer AG, Novartis AG, Biogen Inc., Chugai Pharmaceutical Co. Ltd., Genentech Inc., PTC Therapeutics Inc., Ionis Pharmaceuticals Inc., Biohaven Ltd., Catalyst Pharmaceuticals Inc., RegenXBio Inc., Cytokinetics Inc., AveXis Inc., Scholar Rock Inc., Genethon, CANbridge Pharmaceuticals Inc., NMD Pharma A/S, Hanugen Therapeutics Inc., Beijing Jinlan Gene Technology Co. Ltd.

What Are the Latest Innovations in the Spinal Muscular Atrophy Market?
Key players in the spinal muscular atrophy market are focusing on the development of innovative medical products like dry syrup to enhance treatment adherence and simplify administration among children and other patient groups who struggle with swallowing. Dry syrup, a powder-based medicine mixed with water prior to consumption, is commonly recommended for children and patients who find swallowing tablets or capsules challenging. As an example, in September 2024, Chugai Pharmaceutical Co., Ltd., a Japanese pharmaceutical firm, received regulatory approval from Japan's Ministry of Health, Labour and Welfare to expand the use of its drug Evrysdi (risdiplam). This approval makes Evrysdi applicable to pre-symptomatic spinal muscular atrophy (SMA) patients and extends its usage to infants below two months. This advancement is essential as it allows treatments to begin before the emergence of symptoms, potentially improving therapeutic results for affected infants. The approval was backed by results from the RAINBOWFISH study, examining Evrysdi's safety and effectiveness in infants diagnosed with but not yet exhibiting SMA symptoms. The study's findings indicated that infants who received treatment achieved developmental milestones like independent sitting and improved motor skills within 12 months, highlighting the drug's potential to alter the progression of the disease when used early.

How Is the Segmentation of the Spinal Muscular Atrophy Market Defined?
The spinal muscular atrophy market covered in this report is segmented -

1) By Type: Type 1 (Severe), Type 2 (Intermediate), Type 3 (Mild), Type 4 (Adult)
2) By Age: Pediatric, Adults
3) By Treatment: Drug Therapy, Gene Therapy
4) By End User: Hospital pharmacy, Online pharmacy, Retail pharmacy

Subsegments:
1) By Type 1 (Severe): Infantile-Onset SMA, Severe Progressive SMA
2) By Type 2 (Intermediate): Early-Onset SMA, Non-progressive SMA
3) By Type 3 (Mild): Juvenile-Onset SMA, Mild Progressive SMA
4) By Type 4 (Adult): Adult-Onset SMA, Late-Onset SMA

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What Is the Leading Region in the Spinal Muscular Atrophy Market?
North America was the largest region in the spinal muscular atrophy market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the spinal muscular atrophy market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.

What Is Covered In The Spinal Muscular Atrophy Global Market Report?

- Market Size Analysis: Analyze the spinal muscular atrophy Market size by key regions, countries, product types, and applications.
- Market Segmentation Analysis: Identify various subsegments within the spinal muscular atrophy Market for effective categorization.
- Key Player Focus: Focus on key players to define their market value, share, and competitive landscape.
- Growth Trends Analysis: Examine individual growth trends and prospects in the market.
- Market Contribution: Evaluate contributions of different segments to the overall spinal muscular atrophy market growth.
- Growth Drivers: Detail key factors influencing market growth, including opportunities and drivers.
- Industry Challenges: Analyze challenges and risks affecting the spinal muscular atrophy market.
- Competitive Developments: Analyze competitive developments, such as expansions, agreements, and new product launches in the market.

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