Duchenne Muscular Dystrophy Global Market Report 2024

The duchenne muscular dystrophy market size has grown strongly in recent years. It will grow from $1.07 billion in 2023 to $1.17 billion in 2024 at a compound annual growth rate (CAGR) of 9.2%. The growth observed in the historical period can be attributed to advancements in genetic understanding and diagnostics, progress in clinical trials, increased patient advocacy and awareness, orphan drug designation and incentives, and collaborative research initiatives.

The duchenne muscular dystrophy market size is expected to see strong growth in the next few years. It will grow to $1.61 billion in 2028 at a compound annual growth rate (CAGR) of 8.2%. The anticipated growth in the forecast period can be attributed to the emergence of gene therapies, precision medicine approaches, expanded access to therapies, long-term follow-up studies, and regulatory advancements. Major trends in the forecast period include the integration of digital health technologies, technological advancements in therapeutics, gene therapies, and genetic medicines, as well as regulatory support for orphan drugs and precision medicine approaches.

The growth of the Duchenne muscular dystrophy market is anticipated to be propelled by the increasing prevalence of chronic diseases. Chronic diseases are health conditions with enduring symptoms lasting for at least a year. Duchenne muscular dystrophy plays a role in the prevalence of chronic diseases as it addresses the specific therapeutic needs of individuals with this genetic disorder, underscoring the broader importance of personalized medicine in managing and potentially mitigating the impact of chronic conditions. For instance, as of January 2023, the National Institutes of Health (NIH), a U.S. government agency specializing in biomedical and health-related research, projects a significant increase in the prevalence of chronic diseases among the working-age population in the United States. The estimate indicates a 99.5% rise in adults aged 50 years and older with at least one chronic disease from 2020 to 2035. Moreover, the prevalence of multimorbidity is expected to surge by 110.5% from 2020 to 2050, highlighting the growing health challenges faced by the working population. Therefore, the increasing prevalence of chronic diseases is a key driver for the growth of the Duchenne muscular dystrophy market.

The growth of the Duchenne muscular dystrophy market is anticipated to be propelled by the increasing demand for personalized medicine. Personalized medicine involves tailoring medical treatment to an individual's unique characteristics, such as genetic makeup or lifestyle, leading to more effective and precise healthcare outcomes. In the context of Duchenne muscular dystrophy (DMD), the application of personalized medicine entails customizing treatments based on individual factors, enhancing therapeutic efficacy, and improving patient outcomes. For instance, as of December 2023, a report by The Personalized Medicine Coalition (PMC), a US-based nonprofit organization promoting the use of personalized medicine concepts, services, and products, reveals that personalized medicines constituted 35% of newly approved New Molecular Entities (NMEs) in 2021. They have consistently represented over a quarter of new therapeutics approved since 2015. Over the last six years, personalized medicines have consistently comprised more than a third of new drug approvals. Therefore, the growing demand for personalized medicine is a key driver for the Duchenne muscular dystrophy market's growth.

Technological advancements are a prominent trend gaining traction in the Duchenne muscular dystrophy market. Key companies within this market are intensifying their focus on technological advancements and investing in research and development to fortify their positions. For example, in April 2021, the Hamon Center for Regenerative Science and Medicine at the University of Texas Southwestern, a US-based public academic health science center, introduced an innovative gene therapy approach for treating mice with Duchenne muscular dystrophy (DMD). This approach utilizes CRISPR-Cas9-based techniques to restore a significant portion of the dystrophin protein, which is often deficient in many DMD patients. The researchers developed highly effective CRISPR-Cas9 nucleotide gene editing techniques to override the erroneous stop signal, leading to the restoration of 97% of the protein. This breakthrough involved the exploration of nucleotide gene editing techniques using cardiomyocytes derived from induced pluripotent stem cells (iPS cells) of DMD patients, demonstrating the successful restoration of the dystrophin protein.

Major players in the Duchenne muscular dystrophy market are directing their efforts towards product innovations, notably the development of the first gene therapy to treat Duchenne Muscular Dystrophy (DMD). This innovative therapy involves introducing functional copies of the dystrophin gene into affected individuals, aiming to correct the genetic mutation responsible for the progressive muscle degeneration associated with DMD. For instance, in June 2023, Sarepta Therapeutics, a US-based biopharmaceutical company specializing in RNA-based treatments for rare and infectious diseases, announced the FDA approval of ELEVIDYS, marking a significant milestone as the first gene therapy for Duchenne Muscular Dystrophy. This pioneering one-time treatment employs advanced gene therapy technology to target the root cause of the disease genetically. The approval of ELEVIDYS represents a paradigm shift in DMD treatment, moving beyond traditional supportive care approaches. It holds the potential to enhance patients' quality of life and life expectancy, showcasing the transformative impact of gene therapy on addressing rare diseases.

In December 2022, Solid Biosciences, a US-based life sciences company dedicated to advancing a portfolio of neuromuscular and cardiac programs, acquired AavantiBio for an undisclosed amount. This strategic acquisition positions the combined company to concentrate on the development of a diverse range of neuromuscular and cardiac programs, with a focus on advancing SGT-003, Solid's next-generation gene transfer candidate designed for the treatment of Duchenne muscular dystrophy. AavantiBio, Inc., a US-based biotechnology company, specializes in developing gene therapies aimed at transforming the lives of patients with rare genetic diseases.

Major companies operating in the duchenne muscular dystrophy market report are Pfizer Inc., GlaxoSmithKline plc, Eli Lilly and Company, Daiichi Sankyo Company Limited, PerkinElmer Inc., BioMarin Pharmaceutical Inc., Nippon Shinyaku Co. Ltd., Sarepta Therapeutics Inc., PTC Therapeutics Inc., Acceleron Pharma Inc., FibroGen Inc., Asklepios BioPharmaceutical Inc., Catabasis Pharmaceuticals Inc., Wave Life Sciences Ltd., Bristol Myers Squibb Company, BridgeBio Pharma Inc., CureVac N.V., Rocket Pharmaceuticals Inc., Italfarmaco SpA, Solid Biosciences Inc., Avidity Biosciences Inc., Fulcrum Therapeutics Inc., Akashi Therapeutics Inc., Nihon Pharmaceutical Co. Ltd., WAVE Life Sciences Ltd., Capricor Therapeutics Inc., Santhera Pharmaceuticals Holding, Armagen Technologies Inc., Dyne Therapeutics Inc., Benitec Biopharma Limited, Edgewise Therapeutics Inc.

North America was the largest region in the Duchenne muscular dystrophy market in 2023. Asia-Pacific is expected to be the fastest-growing region in the global duchenne muscular dystrophy market during the forecast period. The regions covered in the duchenne muscular dystrophy market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the duchenne muscular dystrophy market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Duchenne Muscular Dystrophy therapies involve mutation suppression and the exon skipping approach. Exon skipping, a prevalent form of alternative splicing, removes introns and intervals on a pre-mRNA molecule, typically functioning as exons of a gene, when forming the final mRNA. Therapeutic classes include molecular-based therapies and steroid therapy, administered through oral, parenteral, and other routes. These therapies are distributed via hospital pharmacies, online pharmacies, and retail pharmacies, serving end-users such as hospitals, homecare, specialty clinics, and others.

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The duchenne muscular dystrophy market includes revenues earned by entities by providing respiratory therapy, physical therapy and gene therapy. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.