Muscular Dystrophy – Landscape & Forecast – Niche & Rare Disease Landscape & Forecast (US/EU5)

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. The standard treatment for MD is glucocorticoids, which can delay the loss of ambulation. For Duchenne muscular dystrophy (DMD), the most common childhood-onset form, the conditional approvals of disease-modifying therapies (Sarepta’s exon-skipping therapies Exondys 51, Vyondys 53, and Amondys 45; NS Pharma’s Viltepso) reflect uncertainty about these drugs’ efficacy. However, Sarepta’s gene therapy Elevidys and Catalyst / Santhera’s novel corticosteroid Agamree are expanding the treatment options by offering better efficacy. Nevertheless, treatments are still needed that can meaningfully delay or halt the progressive muscle degeneration associated with the various forms of MD. Many therapies with diverse mechanisms of action are in development for DMD and other forms of MD; some of these therapies will help fulfill treatment needs.

Question answered

• What are the sizes of the key MD patient populations in the United States and EU5, and how will they change through 2034?
• How is the gene therapy Elevidys being integrated into the current treatment landscape for DMD?
• What are the greatest unmet clinical needs in the treatment of MD, according to experts? How well will pipeline therapies address current treatment gaps?
• Which emerging therapies will launch by 2034, and what will be their commercial impact on the MD market through 2034?

Content highlights

• Publication date: August 2025
• Geography: United States and EU5
• Primary research: Country-specific interviews with thought-leading neurologists; survey data collected for this and other Clarivate research
• Epidemiology: Diagnosed prevalent and drug-treated cases of DMD, diagnosed prevalent cases of DMD by exon-skipping pattern and ambulatory status, diagnosed prevalent cases of other major MD subtypes such as Becker MD, limb-girdle MD, and myotonic dystrophy
• Forecast: Drug-level sales and patient share of key MD therapies in 2024 and 2034
• Drug treatments: Coverage of key current and late-phase emerging therapies

Product description

Niche & Rare Disease Landscape & Forecast offers comprehensive market intelligence with world-class epidemiology, keen insight into current and emerging therapies, and drug forecasts supported by detailed primary and secondary research, enabling you to:

• Optimize your long-term disease and development strategy.
• Quantify market potential for your pipeline assets and those of your competitors.
• Understand a disease from top to bottom, including key patient populations, the current and future therapeutic landscape, and the evolving market trajectory.
• Gauge the commercial outlook and impact of key market events.