Spinal muscular atrophy (SMA) is a genetic disorder characterized by weakness and wasting (atrophy) of muscles due to the loss of motor neurons in the spinal cord and lower brain stem. It is caused by a mutation in the SMN1 gene and leads to loss of these motor neurons that control muscle movement and strength. Conventional drugs for managing SMA symptoms include glutamine, muscle relaxants, physical therapy, and orthopedic treatment. However, recent advancements in gene therapy for SMA aims at replacing the function of the mutated SMN1 gene and has shown promising results in halting or reversing the disease.

The global spinal muscular atrophy Market is estimated to be valued at US$ 2089.13 Bn in 2023 and is expected to exhibit a CAGR of 7.8% over the forecast period 2024 to 2031, as highlighted in a new report published by Coherent Market Insights.

Market Opportunity:
Gene therapies for SMA hold significant potential for long-term disease modification and present a major market opportunity. Drugs like Zolgensma and Evrysdi have received regulatory approval and commercial success, however, they are limited to treating only infants or children. Several other gene therapy candidates are in clinical trials targeting all SMA subtypes and age groups. With continued research and development, gene therapies are likely to provide a definitive cure for SMA in the future instead of just managing symptoms. This will drive widespread adoption and uptake of novel gene therapies, fueling growth of the global SMA market during the forecast period.

Porter's Analysis
Threat of new entrants: Low as it requires huge R&D cost and capital to develop new drugs for rare disease market.

Bargaining power of buyers: Low as few players operates in the market and buyers have limited options.

Bargaining power of suppliers: Low as suppliers are dependent on few biotech and pharma players.

Threat of new substitutes: Low as there is no substitute for drug treatment available currently.

Competitive rivalry: High among existing players to gain market share as market offers high growth potential.

SWOT Analysis
Strength: Established pipeline of drugs, increasing awareness.

Weakness: High cost of treatment, limited patient pool.

Opportunity: Untapped emerging markets, collaborations.

Threats: Stringent regulations, reimbursement issues.

Key Takeaways
The global Spinal Muscular Atrophy market is expected to witness high growth during the forecast period of 2024 to 2031. The global Spinal Muscular Atrophy Market is estimated to be valued at US$ 2089.13 Bn in 2024 and is expected to exhibit a CAGR of 7.8% over the forecast period 2024 to 2031.

Regional analysis: North America dominates the market currently due to presence of major players and availability of advanced healthcare facilities. The Asia Pacific is expected fastest growing region owing to large patient pool and improving healthcare infrastructure.

Key players operating in the Spinal Muscular Atrophy market are Roche, Novartis, Pfizer and Biogen. Spinal Muscular Atrophy mainly affects children below 2 years. The increased focus of pharmaceutical companies on rare disease is expected to drive market growth in coming years.

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