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Duchenne Muscular Dystrophy (DMD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033 thelansis.com
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.
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 (DMD) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
KOLs insights of Duchenne Muscular Dystrophy (DMD) across 8 MM market from the centre of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.
Duchenne Muscular Dystrophy (DMD) Market Forecast Patient Based Forecast Model (MS. Excel Based Automated Dashboard), which 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.
Thelansis Competitive Intelligence (CI) practice has been established based on a deep understanding of the pharma/biotech business environment to provide an optimized support system to all levels of the decision-making process. It enables business leaders in forward-thinking and proactive decision-making. Thelansis supports scientific and commercial teams in seamless CI support by creating an AI/ ML-based technology-driven platform that manages the data flow from primary and secondary sources.
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