Duchenne Muscular Dystrophy (DMD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Duchenne Muscular Dystrophy (DMD) Market Outlook

Thelansis’s “Duchenne Muscular Dystrophy (DMD) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Duchenne Muscular Dystrophy treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Duchenne Muscular Dystrophy (DMD) Overview

Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by the progressive weakening and wasting of muscles, affecting skeletal, smooth, and cardiac muscle tissues. The underlying cause of muscle damage is the absence of the sarcolemmal protein dystrophin due to variations in the DMD gene located on Xp21.2. Symptoms of DMD typically manifest in early childhood, with affected boys often experiencing delayed walking, typically after 18 months, along with speech and developmental delays. Individuals with DMD may also face autism and behavioral issues, including ADHD, anxiety, and obsessive-compulsive disorder. Unfortunately, untreated children with DMD rarely achieve the ability to run or jump. The condition progresses rapidly, leading to a characteristic waddling gait and a positive Gowers' sign. Climbing stairs becomes challenging, and frequent falls occur. Independent walking is typically lost between the ages of 6 and 13, with an average age of 9.5 in non-steroid treated cases. Once ambulation is lost, joint contractures and scoliosis develop rapidly, and untreated patients typically succumb to respiratory failure or cardiomyopathy in their late teens to early twenties. Diagnosis of DMD is suspected based on clinical presentation, family history, and laboratory findings, particularly elevated serum creatine kinase (CK) levels, often 100-200 times the normal range. Genetic testing is the definitive method and involves multiplex-ligation dependent probe amplification (MLPA) for detecting exon deletions and duplications and full gene sequencing to identify small deletions, duplications, non-sense mutations, or point mutations. Differential diagnoses include severe Becker muscular dystrophy and limb-girdle muscular dystrophy. The gold standard treatment for DMD involves corticosteroids such as prednisolone, prednisone, or deflazacort. These should be initiated early or when the child's motor skills plateau, typically around 4-5 years of age. Despite advancements in management and treatment, DMD has a severe prognosis, significantly reducing life expectancy, with death occurring in the third to fifth decades of life.

• The global prevalence of DMD is estimated at 7.1 cases per 100,000 males and 2.8 cases per 100,000 in the general population. Additionally, the birth prevalence of DMD worldwide stands at 19.8 per 100,000 live male births.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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