| Study Period | 2019 - 2030 |
| Base Year For Estimation | 2024 |
| Forecast Data Period | 2025 - 2030 |
| CAGR | 14.50 % |
| Fastest Growing 乐鱼体育官网 | Asia Pacific |
| Largest 乐鱼体育官网 | North America |
| 乐鱼体育官网 Concentration | Medium |
Major Players
*Disclaimer: Major Players sorted in no particular order |
Spinal Muscular Atrophy Treatment 乐鱼体育官网 Analysis
The Global Spinal Muscular Atrophy Treatment 乐鱼体育官网 is expected to register a CAGR of 14.5% during the forecast period.
Due to strict lockdown measures and restricted movement of people, the COVID-19 virus outbreak has had a significant impact on the spinal muscular atrophy medicine industry. The unprecedented coronavirus outbreak (SARS-CoV-2) has cast a shadow over the SMA treatment industry. For instance, according to the article published in the National Institute of Health in May 2020, titled "Spinal muscular atrophy care in the COVID19 pandemic era," the delay in the SMA treatment during the pandemic affected the delivery of clinical care to patients with spinal muscular atrophy (SMA). Moreover, the amount of money available for the R&D and drug development for SMA has been decreasing, as the majority of the money has been spent on COVID-19 vaccine production.
The major factors accounting for the growth of the spinal muscular atrophy treatment 乐鱼体育官网 are growing awareness regarding SMA, its diagnosis and treatment, rise in the government initiatives to improve healthcare infrastructure, and increasing R&D activities to develop novel treatment regimens. For instance, in August 2020, the US Food and Drug Administration approved Evrysdi (risdiplam) to treat patients two months of age and older with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. This is the second drug and the first oral drug approved to treat this disease. Emerging treatment therapies in the treatment of SMA are driving the growth of the 乐鱼体育官网 during the forecast period.
However, the high cost of treatment and lack of skilled professionals for surgical procedures and physiotherapy regimens hampers the 乐鱼体育官网 to some extent.
Spinal Muscular Atrophy Treatment 乐鱼体育官网 Trends
The Gene Replacement Therapy Segment Dominates the 乐鱼体育官网, and the Same is Expected Over the Forecast Period
Gene replacement therapy is the technique of recognizing a faulty gene, applying a piece of DNA in its correct form through a viral vector (known as the carrier molecule) to the gene, thus, overriding the identified faulty gene with the correct copy.
Gene replacement therapy is expected to dominate the spinal muscular atrophy treatment 乐鱼体育官网 over the forecast period, attributing to its advantages over the disadvantages. However, investment in research and development activities is also expected to have a significant effect on the segment. Companies such as Pfizer Inc. are aiming to build a gene therapy platform with a strategy focused on establishing a transformational portfolio through in-house capabilities and enhancing those capabilities through strategic collaborations, expansion of R&D activities, and potential licensing and merger and acquisition activities. For instance, in August 2019, Pfizer was investing USD 500 million to expand a manufacturing facility in North Carolina, which plays a central role in its efforts to become a major player in the gene therapy industry.
The effects of gene replacement therapy are long-lasting and improve the quality of life by eradicating genetic disorders. Recently in May 2019, the US FDA approved Zolgensma for the treatment of spinal muscular atrophy is a gene therapy, which is found to be the most efficient in results, and many other drugs are in the pipeline. Thus, gene replacement therapy dominates the spinal muscular atrophy treatment 乐鱼体育官网. However, the healthcare cost for gene therapy using Zolgensma is high, but this may hamper the 乐鱼体育官网 to a very less extent, as the government is raising huge funds for the treatment of rare diseases. For example, under the government's Ayushman Bharat scheme in India, the current draft policy provides up to INR 15 lakhs for rare disease treatment to patients who require one-time treatment.
Thus, all aforementioned factors, such as the advantages of gene replacement therapy and technological advancement in gene therapy, may boost the segment over the forecast period.
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North America Dominates the 乐鱼体育官网, and the Same is Expected Over the Forecast Period
North America is expected to dominate the overall 乐鱼体育官网 throughout the forecast period, which is attributed to the developed economy, increasing health care expenditure, increasing incidence of lifestyle-related diseases, and high adoption of technologically advanced products in the region. According to the article published by Columbia University in November 2021, more than 7,000 rare diseases affect 25 million to 30 million American children and adults. More than 90% of rare diseases lack a Food and Drug Administration (FDA)-approved treatment. The reimbursement policies in the United States, especially for the treatment of rare diseases, further boost the 乐鱼体育官网 in North America.
Moreover, many key players in the 乐鱼体育官网 are focused on adopting strategies, such as mergers and acquisitions, to enhance their product portfolio, which, in turn, is expected to propel 乐鱼体育官网 growth over the forecast period. For instance, in August 2020, the US Food and Drug Administration approved Evrysdi (risdiplam) to treat patients two months of age and older with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. Thus, North America dominates the spinal muscular atrophy treatment 乐鱼体育官网 during the forecast period.
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Spinal Muscular Atrophy Treatment Industry Overview
The spinal muscular atrophy treatment 乐鱼体育官网 is moderately competitive and consists of several major players. With the increasing consolidations of various organizations and product recalls in the healthcare sector, it is expected to generate competitive rivalry among the key players in the future. Some of the major players of the 乐鱼体育官网 are Novartis AG, Biogen Inc., F. Hoffmann - La Roche Ltd, and Cytokinetics Inc.
Spinal Muscular Atrophy Treatment 乐鱼体育官网 Leaders
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Biogen
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F. Hoffmann - La Roche Ltd
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Cytokinetics, Inc.,
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Novartis AG
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Catalyst Pharmaceutical
- *Disclaimer: Major Players sorted in no particular order
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Spinal Muscular Atrophy Treatment 乐鱼体育官网 News
- In August 2021, Novartis announced the lift of a partial clinical trial hold and planned to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with spinal muscular atrophy.
- In July 2021, Roche launched prescription medicine Evrysdi in India, which is used for spinal muscular atrophy in adults and children aged two months and older.
Spinal Muscular Atrophy Treatment Industry Segmentation
Spinal muscular atrophy linked to chromosome 5q (SMA) is a recessive, progressive, neuromuscular disorder caused by bi-allelic mutations in the SMN1 gene, resulting in motor neuron degeneration and variable presentation in relation to onset and severity. The spinal muscular atrophy treatment 乐鱼体育官网 is segmented by type (type I, type II, type III, and type IV), procedure (gene replacement therapy and drug therapy), route of administration (oral and intrathecal), and geography (North America, Europe, Asia-Pacific, Middle-East and Africa, and South America). The report offers the value (in USD million) for the above segments. The report offers the value (in USD million) for the above segments.
| By Type | Type 1 | ||
| Type 2 | |||
| Type 3 | |||
| Type 4 | |||
| By Procedure | Gene Replacement Therapy | ||
| Drug Therapy | |||
| Other Procedures | |||
| By Route of Administration | Oral | ||
| Intrathecal | |||
| By Geography | North America | United States | |
| Canada | |||
| Mexico | |||
| Europe | Germany | ||
| United Kingdom | |||
| France | |||
| Italy | |||
| Spain | |||
| Rest of Europe | |||
| Asia-Pacific | China | ||
| Japan | |||
| India | |||
| Australia | |||
| South Korea | |||
| Rest of Asia-Pacific | |||
| Middle-East and Africa | GCC | ||
| South Africa | |||
| Rest of Middle-East and Africa | |||
| South America | Brazil | ||
| Argentina | |||
| Rest of South America | |||
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Spinal Muscular Atrophy Treatment 乐鱼体育官网 Research FAQs
What is the current Global Spinal Muscular Atrophy Treatment 乐鱼体育官网 size?
The Global Spinal Muscular Atrophy Treatment 乐鱼体育官网 is projected to register a CAGR of 14.5% during the forecast period (2025-2030)
Who are the key players in Global Spinal Muscular Atrophy Treatment 乐鱼体育官网?
Biogen, F. Hoffmann - La Roche Ltd, Cytokinetics, Inc.,, Novartis AG and Catalyst Pharmaceutical are the major companies operating in the Global Spinal Muscular Atrophy Treatment 乐鱼体育官网.
Which is the fastest growing region in Global Spinal Muscular Atrophy Treatment 乐鱼体育官网?
Asia Pacific is estimated to grow at the highest CAGR over the forecast period (2025-2030).
Which region has the biggest share in Global Spinal Muscular Atrophy Treatment 乐鱼体育官网?
In 2025, the North America accounts for the largest 乐鱼体育官网 share in Global Spinal Muscular Atrophy Treatment 乐鱼体育官网.
What years does this Global Spinal Muscular Atrophy Treatment 乐鱼体育官网 cover?
The report covers the Global Spinal Muscular Atrophy Treatment 乐鱼体育官网 historical 乐鱼体育官网 size for years: 2019, 2020, 2021, 2022, 2023 and 2024. The report also forecasts the Global Spinal Muscular Atrophy Treatment 乐鱼体育官网 size for years: 2025, 2026, 2027, 2028, 2029 and 2030.
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Statistics for the 2025 Global Spinal Muscular Atrophy Treatment 乐鱼体育官网 share, size and revenue growth rate, created by Mordor Intelligence鈩� Industry Reports. Global Spinal Muscular Atrophy Treatment analysis includes a 乐鱼体育官网 forecast outlook for 2025 to 2030 and historical overview. Get a sample of this industry analysis as a free report PDF download.
