Duchenne muscular dystrophy (DMD) is a severe X-linked recessive neuromuscular disorder causing progressive muscle degeneration. According to Vijay Venugopal et al., 2023, its incidence is 1 in 3,600 male live births, with a U.S. prevalence of 2 per 10,000 males, making it one of the most common and severe congenital myopathies. The therapeutic landscape includes gene therapies, exon-skipping treatments, corticosteroids, and emerging mutation-targeted approaches. According to the Duchenne muscular dystrophy pipeline analysis by Expert Market Research, growing investment in genetic medicine, improved diagnostic capabilities, and expanding clinical trials are expected to drive robust pipeline growth in the coming years.
Major companies involved in the Duchenne muscular dystrophy pipeline analysis include Solid Biosciences Inc., Entrada Therapeutics, Inc., and others.
Leading drugs currently in the pipeline include SGT-003, ENTR-601-45, AOC 1044, and others.
Advancements in gene-editing therapies, rising clinical trial activity for exon-skipping candidates, and increasing investment in mutation-targeted approaches are expected to significantly accelerate pipeline growth in the coming years.
The Duchenne Muscular Dystrophy Pipeline Analysis Report by Expert Market Research gives comprehensive insights into Duchenne muscular dystrophy therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Duchenne muscular dystrophy. The Duchenne muscular dystrophy report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The Duchenne muscular dystrophy pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with Duchenne muscular dystrophy treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to Duchenne muscular dystrophy.
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Duchenne muscular dystrophy is a severe genetic disorder caused by mutations in the DMD gene, resulting in the absence of dystrophin and progressive muscle degeneration. It primarily affects boys, with symptoms such as difficulty walking and frequent falls appearing in early childhood and gradually worsening as the disease advances.
Duchenne muscular dystrophy treatments aim to slow muscle deterioration through corticosteroids, structured physical therapy, mobility aids, and emerging genetic therapies that target dystrophin mutations. In June 2023, the treatment landscape progressed as Elevidys showed promise by increasing micro-dystrophin expression in early evaluations, supporting the development of more effective gene-based therapies for eligible pediatric patients.
Duchenne muscular dystrophy (DMD) drug pipeline analysis shows important epidemiological trends. According to Vijay Venugopal et al., the incidence is 1 in 3,600 male live births, with a prevalence of 2 per 10,000 in the United States. As per Xin Wang et al., the incidence reaches 119.50 per 100,000 males in the United Kingdom. Taiho Pharmaceutical Co., Ltd. reported a prevalence of 1.9–3.4 per 100,000 individuals in Japan. These findings highlight the ongoing need for effective therapeutic progress.
This section of the report covers the analysis of Duchenne muscular dystrophy drug candidates based on several segmentations, including:
By Phase
The pipeline assessment report covers 50+ drug analyses based on phase:
By Drug Class
The Duchenne muscular dystrophy pipeline analysis report covers 50+ drug analyses based on drug classes:
By Route of Administration
The pipeline assessment report covers 50+ drug analyses based on the route of administration.
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase II at 45.07%, covers a major share of the total Duchenne muscular dystrophy clinical trials. It is followed by phase I at 24% and phase III at 20%. The early phase I stands at 7%. This balanced distribution across phases reflects strong research progress, supporting continuous innovation and strengthening future growth opportunities in the Duchenne muscular dystrophy market.
The drug molecule categories covered under the Duchenne muscular dystrophy pipeline analysis include small molecules, monoclonal antibodies, cell therapies, peptides, and polymers. The Duchenne muscular dystrophy report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Duchenne muscular dystrophy. Cell-based therapies are gaining prominence in the Duchenne muscular dystrophy drug pipeline as they aim to address both skeletal and cardiac muscle deterioration. For example, Deramiocel, an allogeneic cardiosphere-derived cell therapy, has demonstrated clinically meaningful benefits by slowing disease progression and preserving cardiac function, positioning it as a promising first-in-class therapeutic option.
The EMR report for the Duchenne muscular dystrophy pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed Duchenne muscular dystrophy therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in Duchenne muscular dystrophy clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for Duchenne muscular dystrophy. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of Duchenne muscular dystrophy drug candidates.
SGT-003 is an investigational gene therapy sponsored by Solid Biosciences Inc., designed to deliver a differentiated microdystrophin construct via a single intravenous infusion. The Phase 3 study aims to evaluate its efficacy and safety in ambulant males with Duchenne muscular dystrophy. The therapy is using the next-generation AAV-SLB101 capsid, engineered to target integrin receptors and enhance cardiac and skeletal muscle transduction while reducing liver exposure. The study is examining how SGT-003 restores microdystrophin expression, improves muscle function, and potentially reduces muscle fatigue by localizing nNOS to muscle tissue.
ENTR-601-45, sponsored by Entrada Therapeutics, Inc., is being evaluated in this Phase 1/2 study to assess its safety, tolerability, and early efficacy in Duchenne muscular dystrophy patients amenable to exon 45 skipping. The study is examining how this EEV™-conjugated PMO is entering muscle cells more efficiently, escaping endosomes, and binding to the DMD pre-mRNA to promote exon 45 skipping. By restoring the mRNA reading frame, ENTR-601-45 is enabling the body to produce a shortened but functional dystrophin protein. The drug is being administered through intravenous infusions.
AOC 1044 is being sponsored by Avidity Biosciences, Inc. and is currently being evaluated in an active, not-recruiting Phase 2 open-label extension study for Duchenne muscular dystrophy. The study is examining the long-term safety, tolerability, and pharmacodynamics of this antibody-oligonucleotide conjugate, which delivers PMOs to skeletal and cardiac muscle to enable exon 44 skipping and restore dystrophin production. AOC 1044 is administered intravenously every six weeks and continues to demonstrate targeted delivery via its TfR1-binding monoclonal antibody design.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The Duchenne Muscular Dystrophy Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for Duchenne muscular dystrophy. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into Duchenne muscular dystrophy collaborations, regulatory environments, and potential growth opportunities.
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*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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