Spinal Muscular Atrophy Treatment Market: Major Trends Reshaping the Future of the Industry

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What Will the Spinal Muscular Atrophy Treatment Industry Market Size Be by 2025?
The market size of treatments for spinal muscular atrophy has seen a swift expansion in the past few years. It is expected to increase from $4.41 billion in 2024 to $5.1 billion in 2025, with a compound annual growth rate (CAGR) of 15.7%. The surge during the historical period can be linked to a rise in the occurrence of spinal muscular atrophy, an increase in approvals for innovative gene therapies, heightening awareness for early diagnosis and treatment, an escalation in research and development investments, and the growing availability of neonatal screening programs.

What's the Long-Term Growth Forecast for the Spinal Muscular Atrophy Treatment Market Size Through 2029?
Anticipated to witness substantial growth in the coming years, the market size for treatment of spinal muscular atrophy is on a trajectory to reach$9.02 billion by 2029, expanding at a compound annual growth rate (CAGR) of 15.3%. The growth anticipated in the forecasted period can be linked to factors such as an increased prevalence of spinal muscular atrophy, more approvals for innovative gene therapies, heightened cognizance of early diagnosis and treatment, an influx in investments for research and development, and the escalating accessibility of newborn screening programs. The forecasted period is likely to see major trends such as advancements in gene therapy techniques, integration of personalized medicine approaches, the utilization of technology in developing oral therapies, progress in newborn screening programs, and advancements in combination therapy strategies.

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What Are the Key Growth Drivers Fueling the Spinal Muscular Atrophy Treatment Market Expansion?
The emphasis on gene therapy is anticipated to boost the expansion of the spinal muscular atrophy treatment market. Gene therapy is a medical procedure that modifies an individual's DNA or RNA in order to treat or prevent illness. The escalating attention towards gene therapy is mainly because of its capacity to directly modify or substitute malfunctioning genes, effectively addressing the underlying problems of genetic disorders, presenting longer-term or even permanent treatment outcomes compared to conventional approaches. Treatments like onasemnogene abeparvovec (Zolgensma) for spinal muscular atrophy demonstrate the benefits of gene therapy, as they provide a single, direct method that supplies a functional version of the SMN1 gene, substantially enhancing motor function and life quality for SMA patients. For example, in January 2024, the American Society of Gene and Cell Therapy, a US-based gene and cell therapy entity, reported that the number of gene therapies in Phase III trials grew by 10% in the last quarter of 2023, compared to the former quarter. This signified the initial such growth since the third quarter of 2022. Consequently, the intensified emphasis on gene therapy is stimulating the advancement of the spinal muscular atrophy treatment market.

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What Are the Key Trends Driving Spinal Muscular Atrophy Treatment Market Growth?
Leading firms in the spinal muscular atrophy treatment industry are putting their efforts into conceiving revolutionary solutions like survival motor neuron 2 (SMN2) splicing modifier tablets. These are engineered to augment motor function and decelerate the progression of the disease. The tablets are oral drugs crafted to modify the SMN2's gene's splicing, facilitating the generation of useful SMN protein, essential for motor neuron preservation. The tablets work to alleviate spinal muscular atrophy (SMA) by advancing the levels of full-length SMN protein, leading to enhanced muscle function and a slowdown in disease progression. For instance, Genentech, Inc., a biotechnology corporation based in the US, obtained U.S. Food and Drug Administration endorsement for its Evrysdi (risdiplam) tablet, the pioneer and solitary non-invasive SMN2 splicing modifier tablet for treating SMA, in February 2025. This Evrysdi tablet provides comparable effectiveness and safety, mirroring the existing oral solution, but with added perks like room-temperature storage and simplified administration for patients aged 2 years and above who weigh upwards of 44 lbs (20 kgs), thus offering increased convenience and adaptability in managing the disease.

How Is the Spinal Muscular Atrophy Treatment Market Segmented?
The spinal muscular atrophy treatment market covered in this report is segmented -

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

Subsegments:
1) By Medication: Nusinersen (Spinraza), Onasemnogene Abeparvovec-Xioi (Zolgensma), Risdiplam (Evrysdi), Antisense Oligonucleotides, Spinal Muscular Atrophy (SMN2) Splicing Modifiers, Corticosteroids, Supportive Medications
2) By Physical Therapy: Occupational Therapy, Respiratory Therapy, Aquatic Therapy, Orthotic Support, Assistive Mobility Devices, Range-Of-Motion Exercises
3) By Surgery: Spinal Fusion Surgery, Scoliosis Correction Surgery, Orthopedic Surgery, Gastrostomy Tube Placement, Tracheostomy
4) By Other Treatments: Nutritional Support, Respiratory Support, Psychosocial Support And Counseling, Genetic Counseling, Stem Cell Therapy

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Which Companies Are Leading the Charge in Spinal Muscular Atrophy Treatment Market Innovation?
Major companies 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., Ionis Pharmaceuticals Inc., Ultragenyx Pharmaceutical Inc., Catalyst Pharmaceuticals Inc., BIOCAD INDIA PRIVATE LIMITED, Cytokinetics Incorporated, Voyager Therapeutics Inc., Scholar Rock Holding Corporation, Genethon, Exegenesis Bio Inc, NMD Pharma A/S, Biohaven Pharmaceutical Holding Company Ltd, Alcyone Therapeutics Inc., Vybion Inc., Apteeus SAS, Biophytis SA, Ractigen Therapeutics.

Which Regions Are Leading the Global Spinal Muscular Atrophy Treatment Market in Revenue?
North America was the largest region in the spinal muscular atrophy treatment 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 treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.

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