The Duchenne Muscular Dystrophy (DMD) Therapeutics Market is expected to grow at a CAGR of 28.21% to reach USD 4373.3 million by 2028. The global Duchenne Muscular Dystrophy (DMD) therapeutics market represents a significant business opportunity due to the limited treatment options available for this progressive indication. Multiple pharmaceutical vendors, both large and mid-sized, are actively investing in research and development of novel therapeutics, including antisense oligonucleotide-based therapy, gene therapy, cell therapy, biologics, and small molecule-based drugs. The unmet medical need for effective DMD treatments is driving this market growth. For instance, in February 2021, Sarepta Therapeutics received FDA approval for AMONDYS 45 (casimersen) injection, marking a significant milestone in the treatment landscape for DMD patients amenable to skipping Exon 45. This approval underscores the potential of innovative therapeutic approaches in addressing the unmet medical need in this indication.
North America dominates the regional market because of The Duchenne Muscular Dystrophy (DMD) therapeutics market represents a significant business opportunity for pharmaceutical companies, driven by the high unmet medical need and the potential for innovative treatments. With the increasing understanding of the genetic basis of DMD and advances in gene therapy, the market is expected to experience robust growth in the coming years. Strategic investments in research and development, as well as collaborations and partnerships, will be key to capitalizing on this opportunity.
There are various factors that drive the Duchenne Muscular Dystrophy (DMD) Therapeutics Market market which are as following: Duchenne Muscular Dystrophy (DMD) is a prevalent and severe form of muscular dystrophy, characterized by progressive muscle weakening and atrophy, leading to premature death. This hereditary childhood myopathy affects the skeletal muscles, causing significant impairment. Currently, pharmaceutical interventions for DMD primarily focus on symptomatic management. However, advancements in research have led to the identification of the dystrophin gene, which is responsible for producing proteins essential for muscle cell membrane stability. Animal models, including those with natural mutations and gene-targeted variants, have significantly advanced our understanding of DMD's etiology and disease progression. This knowledge has paved the way for the development of promising therapeutic targets, such as dystrophin-based therapies like EXONDYS 51 and TRANSLARNA. These innovative approaches aim to address the underlying cause of DMD, offering hope for effective treatments and improved patient outcomes.
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Duchenne Muscular Dystrophy (DMD) Therapeutics Market Segmentation
Type (Biologics, Small molecules) Distribution Channel (Offline, Online) Geography (North America, Europe, Asia, Rest of World (ROW))
The market witnesses several challenges, which are as follows Duchenne Muscular Dystrophy (DMD) is a debilitating and fatal genetic condition primarily impacting the male population. This disorder, characterized by muscle weakness, affects children as early as age five, leading to loss of ambulation by ten years and eventual paralysis. The disease burden is significant due to the progressive muscle deterioration, which can result in respiratory and heart failure, ultimately causing premature death. DMD is caused by the deficiency of functional dystrophin protein, a crucial component for maintaining structural integrity of skeletal and heart muscles. The lack of this protein results in muscle degeneration, causing severe disability and significantly impacting patients' and caregivers' quality of life.
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Some of the key companies that have been profiled in the report include:
The Duchenne Muscular Dystrophy (DMD) Therapeutics Market is a significant segment of the global pharmaceuticals industry, encompassing organizations involved in the research and development (R&D) or production of various drug classes, including generic and non-generic medications, as well as veterinary drugs. According to Technavio, the global pharmaceuticals market size is determined by the consolidated revenue generated by manufacturers and providers of equipment, supplies, pharmaceuticals, biotechnology, and life sciences tools and services. Key growth drivers for this market include the increasing global population aging, with the proportion of individuals over 60 years old in the United States projected to reach approximately 25% by 2050, and Europe projected to reach a similar population ratio by 2030. This demographic shift will lead to a substantial increase in demand for pharmaceutical products to address the healthcare needs of an aging population. - The Duchenne Muscular Dystrophy (DMD) Therapeutics Market is experiencing significant growth, fueled by the High unmet need for duchenne muscular dystrophy therapeutics. Businesses are leveraging the products belonging to the market for customer engagement, transactional notifications, and promotional offers.
Duchenne Muscular Dystrophy (DMD) is a progressive genetic disease affecting approximately 1 in every 3,500 live births. The DMD treatment market is experiencing sustainable growth due to strategic business decisions by key players and advancements in therapeutic approaches. Portland, Oregon, is home to several academic institutions and research organizations, making it a hub for DMD research. Investment pockets in the DMD treatment market include steroid therapy segment, exon skipping drugs, molecular-based therapies segment, and gene replacement therapies. Nonsense mutation-specific therapies like Translarna (ataluren) by Sarepta Therapeutics and Emflaza by BioMarin have shown clinical efficacy. SANTHERA PHARMACEUTICALS' Elamipretide, Tabrecta, and Sarepta's Eteplirsen are under investigation for DMD treatment. Strong collaborations between pharmaceutical companies and research organizations are driving gene therapy advancements, including myostatin inhibition and exon-skipping platforms. Orphan Drug Designation has been granted to several DMD therapies, including fordadistrogene movaparvovec by Sarepta Therapeutics. The hospital pharmacies segment is expected to dominate the market due to the high demand for specialized therapies for ambulatory children with DMD. Despite these advancements, the DMD treatment market faces stringent regulatory procedures, making it essential for companies to focus on clinical trial success and safety data to secure approvals. The market is expected to continue growing as new therapies enter the market and existing ones gain wider adoption.
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