Muscular dystrophy (MD) is a spectrum of genetic disorders characterized by muscle weakness that, in severe forms, can lead to loss of ambulation and early mortality. There is a high unmet need for effective treatments that can meaningfully delay or halt progressive muscle degeneration in Duchenne muscular dystrophy (DMD), the most common childhood-onset form, as well as in other forms of MD. Ataluren (PTC Therapeutics’ Translarna), which targets a genetically defined subset of DMD patients, is approved for DMD in Europe; eteplirsen (Sarepta’s Exondys 51), which targets a different genetically distinct DMD subgroup, and deflazacort (PTC Therapeutics’ Emflaza), which is a glucocorticoid, have garnered regulatory approval for DMD in the United States. Additionally, many therapies with diverse mechanisms of action are being developed to treat other forms of MD, including limb-girdle MD and Becker MD. Although therapies with disease-modifying potential have reached the DMD market, there is still a high unmet need for additional and, ideally, more-effective medications for DMD and other forms of MD.

Table of contents

  • Disease Landscape & Forecast
    • COVID-19
    • Commercial Outlook
      • Key Findings
      • Drivers and Constraints
        • What Factors Are Driving the Market for Muscular Dystrophy?
        • What Factors Are Constraining the Market for Muscular Dystrophy?
      • Drug-Class-Specific Trends
        • Gene Therapy-Specific Trends
        • Exon Skipping Therapy-Specific Trends
        • Nonsense Read-Through Therapy
        • Glucocorticoids
        • Antioxidants
        • Histone Deacetylase Inhibitors
    • Forecast
    • Etiology and Pathophysiology
      • Disease Overview
      • Etiology
        • Genetic Causes of Muscular Dystrophies
        • Dystrophin Mutations Underlying Duchenne and Becker Muscular Dystrophy
        • Limb-Girdle and Congenital Muscular Dystrophy
        • Myotonic Dystrophy
      • Pathophysiology
        • Duchenne and Becker Muscular Dystrophy
        • Limb-Girdle and Congenital Muscular Dystrophy
        • Myotonic Dystrophy
      • Biomarkers for Duchenne Muscular Dystrophy
      • Disease Onset and Progression
        • Duchenne and Becker Muscular Dystrophy
        • Limb-Girdle and Congenital Muscular Dystrophy
        • Myotonic Dystrophy
      • Key Pathways and Drug Targets
    • Epidemiology
      • Key Findings
      • Epidemiology Populations
        • Diagnosed Prevalent Cases of Duchenne Muscular Dystrophy
        • Drug-Treatable Cases of Duchenne Muscular Dystrophy
        • Diagnosed Prevalent Cases of Duchenne Muscular Dystrophy by Exon-Skipping Pattern
        • Diagnosed Prevalent Cases of Duchenne Muscular Dystrophy by Ambulatory Status
        • Diagnosed Prevalent Cases of Becker Muscular Dystrophy
        • Diagnosed Prevalent Cases of Limb-Girdle Muscular Dystrophy
    • Current Treatment
      • Key Findings
      • Diagnosis
        • Genetic Testing for Duchenne Muscular Dystrophy
        • Newborn Screening for Duchenne Muscular Dystrophy
        • Treatment Providers and Referral Patterns
      • Treatment Goals
        • The Six-Minute Walk Test
      • Key Current Therapies
        • Glucocorticoids
        • Exon Skipping Therapy
        • Nonsense Read-Through Therapy
        • Key Therapies for Symptomatic Management of Muscular Dystrophy
      • Medical Practice
        • Multidisciplinary Management of Muscular Dystrophy
        • Region-Specific Treatment
    • Unmet Need Overview
      • Current and Future Attainment of Unmet Needs in Muscular Dystrophy
    • Emerging Therapies
      • Key Findings
      • Key Emerging Therapies
        • Exon Skipping Therapies
        • Antioxidants
        • Histone Deacetylase Inhibitors
        • Dystrophin-Based Gene Therapies
      • Early-Phase Pipeline Analysis
      • Patient Registries
        • Patient Organizations
      • Orphan-Drug Designation
        • Orphan-Drug Provisions: United States
        • Orphan-Drug Provisions: Europe
    • Access and Reimbursement Overview
      • Region-Specific Reimbursement Practices
        • United States
        • EU5
    • Methodology
      • Bottom-Up Market Forecasting Overview
        • Patient Populations
        • Drug-Specific Assumptions
      • Bottom-Up Forecast Assumptions
        • Drug-Treatable Rate Assumptions in Muscular Dystrophy
        • General Statements About Pricing
        • Dosing, Days of Therapy, and Compliance
        • Generic Erosion
        • Out-Year Forecasting
        • Emerging Therapy Prices
      • Primary Market Research
        • Experts Interviewed
    • Appendix
      • Muscular Dystrophy Bibliography

Author(s): Pallavi Rajput, Ph.D; Shilpa Thakur

Pallavi Rajput joined Decision Resources Group as an associate analyst in 2019. Her focus is on the disease landscape and forecast of infectious diseases, niche and rare diseases. She holds a in molecular biology and virology from the International Centre for Genetic Engineering and Biotechnology, New Delhi, India. Prior to joining Decision Resources Group, Pallavi was a postdoctoral fellow at the University of California- Davis, where her research focused on the role of DNA repair in cancer development.

Shilpa Thakur is a medical graduate with a from the Postgraduate Institute of Medical Education and Research with a specialization in epidemiology and biostatistics. She specializes in developing epidemiological forecasts for the multiple indications within the DRG syndicated portfolio. Prior to joining Decision Resources, she monitored HIV sentinel surveillance 2016-2017 in Himachal Pradesh. She also has worked on to see the patterns of Antimicrobial resistance in India.  


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