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Research & Reports

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Muscular Dystrophy | Landscape & Forecast | Disease Landscape & Forecast

Muscular Dystrophy | Landscape & Forecast | Disease Landscape & Forecast

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Muscular dystrophies (MDs) are a spectrum of genetic disorders characterized by muscle weakness that, in severe disease forms, can lead to loss of ambulation and early death. Duchenne muscular dystrophy (DMD) is the most common subtype with childhood-onset, while myotonic dystrophy is the most common subtype with adult-onset. The unmet need for effective treatments that can meaningfully delay or halt progressive muscle degeneration in DMD, as well as in other severe and moderately severe MDs, is high. Ataluren (PTC Therapeutics’ Translarna), which targets DMD patients with nonsense dystrophin mutations, has been available in some European markets for DMD following its conditional approval by the EMA in 2014; and eteplirsen (Sarepta’s Exondys 51), which targets DMD patients with dystrophin mutations amenable for skipping of exon-51, has been available in the United States since 2016, when it was granted conditional approval for DMD. Though both drugs target the underlying cause of disease in DMD and, thus, could modify its course, their efficacy as observed in clinical trials appears modest. We anticipate the launch of two new potentially disease-modifying therapies—Sarepta Therapeutics’ exon-skipping agents golodirsen and casimersen—and two nonspecific symptomatic therapies—Santhera Pharmaceuticals’ idebenone and Italfarmaco SpA’s givinostat—during the 2017-2027 forecast period. Despite the launch of four new agents, the unmet need for effective pharmacotherapies for DMD will remain high.

Table of contents

  • Disease Landscape & Forecast
    • 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
        • Exon-Skipping Therapies
        • Nonsense Read-Through Therapy
        • Glucocorticoids
        • Antioxidants
        • Histone Deacetylase Inhibitors
      • Alternative Market Scenarios
    • Forecast
    • Etiology and Pathophysiology
      • Disease Overview
      • Etiology
        • Dystrophin Mutations Underlying Duchenne and Becker Muscular Dystrophy
        • Limb-Girdle Muscular Dystrophy and Congenital Muscular Dystrophy
        • Myotonic Dystrophy
        • Classification of Muscular Dystrophies Based on Gene Mutations
      • Pathophysiology
        • Dystrophin
        • The Dystrophin-Associated Protein Complex
        • Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
        • Limb-Girdle Muscular Dystrophy and Congenital Muscular Dystrophy
        • Myotonic Dystrophy
      • Biomarkers for Duchenne Muscular Dystrophy
      • Disease Onset and Progression
        • Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
        • Limb-Girdle Muscular Dystrophy and Congenital Muscular Dystrophy
        • Myotonic Dystrophy
      • Key Pathways and Drug Targets
        • Antisense Oligonucleotide-Mediated Exon Skipping
        • Nonsense Read-Through
    • Epidemiology Overview
      • Key Findings
      • Epidemiology Populations
        • Diagnosed Prevalent Cases of Duchenne Muscular Dystrophy
        • Drug-Treated 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 Overview
      • Key Findings
      • Diagnosis
        • Genetic Testing for Duchenne Muscular Dystrophy
        • Newborn Screening for Duchenne Muscular Dystrophy
        • Treatment Providers and Referral Patterns
      • Treatment Goals
        • Key End Points Used in Clinical Trials for Muscular Dystrophy
        • The Six-Minute Walk Test
      • Key Current Therapies
        • Overview
        • Glucocorticoids
        • Exon-Skipping Therapies
        • Nonsense Read-Through Therapy
        • Key Therapies for Symptomatic Management of Muscular Dystrophy
      • Medical Practice
        • Overview
        • Treatment Guidelines
        • Multidisciplinary Management of Muscular Dystrophy
        • Drug Selection
        • Region-Specific Treatment
    • Unmet Need Overview
      • Current and Future Attainment of Unmet Needs in Muscular Dystrophy
    • Emerging Therapies Overview
      • Key Findings
      • Key Emerging Therapies
        • Notable Developments Among Key Emerging Therapies for Muscular Dystrophy
        • Exon-Skipping Therapies
        • Antioxidants
        • Histone Deacetylase Inhibitors
        • Dystrophin-Based Gene Therapies
      • Early-Phase Pipeline Analysis
        • Notable Developments in the Early-Phase Pipeline for Muscular Dystrophy
      • Development Discontinuations and Failures in Muscular Dystrophy
      • 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-Treatment Rate Assumptions in Duchenne Muscular Dystrophy
        • General Statements About Pricing
        • Out-Year Forecasting
        • Emerging Therapy Prices
      • Primary Market Research
        • Experts Interviewed
    • Appendix
      • Muscular Dystrophy Bibliography

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  • Pub Date: October 2018
  • Author(s): Akash Saini, PhD; Amy Bradshaw Kaiser, MS
  • Akash Saini, is a lead analyst with the Infectious, Niche, and Rare Disease team at Decision Resources Group. Prior to joining Decision Resources Group, Saini was a postdoctoral fellow at the University of Massachusetts Medical School, where he studied mitochondrial dysfunction in amyotrophic lateral sclerosis (ALS). He earned a in Biochemistry and Biotechnology from the International Centre for Genetic Engineering and Biotechnology, New Delhi, where he also studied the underlying disease mechanism in ALS, and an in Biotechnology from Jawaharlal Nehru University, New Delhi, where he studied the biophysical characteristics of amyloid formation.

  • Amy Kaiser joined Decision Resources Group as an associate epidemiologist in 2017. Her focus is on the epidemiology of infectious diseases and niche and rare diseases. She holds an MS in Epidemiology from the University of Massachusetts, Amherst and a BA in International Relations from Mount Holyoke College. Prior to joining Decision Resources Group, she worked as a human health research associate at a environmental consulting firm where her epidemiology research focused on occupational and environmental exposures and associated outcomes.

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