Number of pages: 100 | Report Format: PDF | Published date: February 23, 2023
Historical Years – 2020 | Base Year – 2021 | Forecasted Years – 2022-2030
Report Attribute |
Details |
Market Size Value in 2021 |
US$ 942.45 million |
Revenue Forecast in 2030 |
US$ 2883.4 million |
CAGR |
13.23% |
Base Year for Estimation |
2021 |
Forecast Period |
2022-2030 |
Historical Year |
2020 |
Segments Covered |
Treatment, Distribution Channel, and Region |
Regional Scope |
North America, Europe, Asia Pacific, and the Rest of the World (RoW) |
According to the deep-dive market assessment study by Growth Plus Reports, the global Duchenne muscular dystrophy treatment market was valued at US$ 942.45 million in 2021 and is expected to register a revenue CAGR of 13.23% to reach US$ 2883.4 million by 2030.
Duchenne Muscular Dystrophy Treatment Market Fundamentals
Duchenne muscular dystrophy (DMD) is a rare genetic disorder primarily affecting males. It is caused by a mutation in the gene that codes for the protein dystrophin, which is necessary for muscle function. Without dystrophin, muscle cells become damaged and eventually die, leading to progressive muscle weakness and loss of function. Symptoms of DMD typically appear in early childhood and progressively worsen over time, eventually leading to the loss of the ability to walk and requiring the use of a wheelchair. Currently, there is no cure for DMD; however, several treatments are available to manage symptoms and slow down the progression of the disease. These treatments include corticosteroids, which can help to improve muscle strength and delay the loss of muscle function, as well as physical therapy and other supportive measures.
Symptoms of DMD typically appear in early childhood and include difficulty walking, frequent falls, and difficulty getting up from a seated or lying position. As the disease progresses, it can lead to loss of mobility, difficulty in breathing, and eventually heart failure. Recent advances in gene therapy and other emerging treatments offer hope for more effective treatments for DMD in the future.
However, there are significant challenges to be addressed in developing and implementing these treatments, including ensuring their safety and efficacy and addressing issues of affordability and access.
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Duchenne Muscular Dystrophy Treatment Market Dynamics
The increasing prevalence of DMD, a genetic disorder that primarily affects young boys, is a key factor driving the market. According to a March 2021 article by the National Organization for Rare Disorders, DMD is one of the most common genetic disorders, affecting around 1 in every 3,500 male births globally. It is commonly identified between the ages of 3 and 6. There are currently several promising drugs in the pipeline, including gene therapy and exon-skipping therapies, which have the potential to slow the progression of the disease and improve patient outcomes. The development of these new treatments is expected to drive growth opportunities in the global Duchenne muscular dystrophy treatment market.
Growing awareness about DMD is leading to increased funding for research and development of new treatments. Governments, patient organizations, and pharmaceutical companies are investing in the development of new therapies for the disease, fueling the revenue growth of the global Duchenne muscular dystrophy treatment market.
However, stringent regulatory approvals, lack of standardized procedures to determine the clinical efficacy of drugs, and the high cost of therapeutics are some of the restraints impeding the overall market revenue growth.
Duchenne Muscular Dystrophy Treatment Market Ecosystem
The global Duchenne muscular dystrophy treatment market is analyzed from three perspectives: therapeutics, distribution channel, and region.
Duchenne muscular dystrophy treatment Market by Therapeutics
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Based on the therapeutics, the global Duchenne muscular dystrophy treatment market is segmented into molecular therapies, steroid therapy, non-steroidal anti-inflammatory drugs (NSAIDs), and others.
The molecular therapies segment dominated the market with the principal revenue share in 2021, owing strong product pipeline and a rise in the number of product launches. This segment includes a range of therapies designed to target the underlying genetic mutations that cause the disease. There are several types of molecular therapies, including exon-skipping therapy and gene therapy. Among these, exon-skipping therapy is one of the most promising molecular-based therapies for DMD. This therapy is designed to skip over the gene’s parts containing the mutation and produce a shorter but functional form of the protein. This therapy can potentially slow the disease’s progression and improve patient outcomes. Several exon-skipping therapies are currently in development, including eteplirsen and golodirsen, which have been approved for use in the U.S.
Gene therapy, another molecular-based therapy for DMD, involves introducing a functional copy of the dystrophin gene into the patient’s cells through viral vectors or gene editing techniques. This therapy has shown promising results in preclinical studies, and several clinical trials are currently underway to evaluate its safety and efficacy. These factors contribute to the revenue growth of the molecular therapies segment.
Duchenne Muscular Dystrophy Treatment Market by Distribution Channel
Based on the distribution channel, the global Duchenne muscular dystrophy treatment market is segmented into hospital, retail, and online pharmacy.
The hospital pharmacy segment dominated the market with the largest revenue share in 2021, owing to the large footfall of patients. Hospital pharmacies are involved in the distribution and administration of several types of medications, including corticosteroids, which are used to slow down the progression of the disease and improve muscle strength, and various molecular-based therapies, such as exon-skipping therapies and gene therapies. Hospital pharmacies also play a critical role in the management of patient care, working closely with other healthcare providers to develop treatment plans and ensure that patients receive comprehensive care. They may also provide patient education and support services to help patients and their families manage the challenges of living with DMD. These overarching variables contribute to the hospital pharmacy segment revenue growth.
Duchenne Muscular Dystrophy Treatment Market by Region
Geographically, the global Duchenne muscular dystrophy treatment market has been segmented into North America, Europe, Asia Pacific, and the Rest of the World.
North America dominated the global Duchenne muscular dystrophy treatment market with the largest revenue share in 2021, attributed to the presence of key players and high awareness about the treatments related to DMD. Additionally, partnerships, mergers, and acquisitions between major firms across the region are also helping in the revenue growth of the regional market.
Duchenne Muscular Dystrophy Treatment Market Competitive Landscape
The prominent players operating in the global Duchenne muscular dystrophy treatment market are:
Duchenne Muscular Dystrophy Treatment Market Strategic Developments
The global Duchenne muscular dystrophy treatment market is expected to be valued at US$ 2,883.4 million by 2030.
The Duchenne muscular dystrophy treatment market is expected to register a revenue CAGR of 13.23% during the forecast period from 2022 to 2030.
The increasing prevalence of DMD and growing funding for research and the development of new treatments and promising drugs in the pipeline are driving the revenue growth of the global Duchenne muscular dystrophy treatment market.
The molecular therapies segment dominates the global Duchenne muscular dystrophy treatment market.
Pfizer Inc., Sarepta Therapeutics, Inc., Santhera Pharmaceuticals, PTC Therapeutics, and Nobelpharma Co., Ltd. are among the top market players.
*Insights on financial performance are subject to the availability of information in the public domain