Muscular Dystrophy | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2024

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. Treatments are needed that can meaningfully delay or halt the progressive muscle degeneration associated with Duchenne muscular dystrophy (DMD), the most common childhood-onset form, as well as other forms of MD. The standard treatment for DMD is glucocorticoids, which have proven effective in delaying the loss of ambulation. Conditional approvals of disease-modifying therapies (Sarepta’s exon-skipping therapies Exondys 51, Vyondys 53, and Amondys 45; and NS Pharma’s Viltepso) reflect uncertainty about these drugs’ clinical efficacy. However, new therapies, such as Sarepta’s gene therapy Elevidys, will provide more treatment options with improved efficacy. Additionally, many therapies with diverse mechanisms of action are in development for other forms of MD, including limb-girdle MD and myotonic dystrophy. Overall, the need for more-effective treatments for MD remains high.

Question answered

• What are the sizes of the key MD patient populations in the United States and EU5, and how will these populations change through 2033?
• What is the current treatment landscape for MD, and what are the greatest unmet clinical needs according to experts?
• How well will pipeline therapies address current treatment gaps?
• Which emerging therapies will launch before 2033, and what will be their commercial impact on the MD market through 2033?

Content highlights

• Geographies: United States and EU5
• Primary research: Six country-specific interviews with thought-leading neurologists supported by survey data collected for this study
• 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 muscular dystrophy therapies through 2033
• Coverage of key current and emerging therapies

Product description

Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.

Key feature

Niche & Rare Disease Landscape & Forecast features a Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.