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 exon-skipping therapies (Sarepta’s Exondys 51, Vyondys 53, and Amondys 45; NS Pharma’s Viltepso) reflect uncertainty about these drugs’ efficacy. Other therapies with disease-modifying potential, such as Sarepta’s gene therapy Elevidys, could offer greater efficacy in ambulatory DMD patients; Catalyst / Santhera’s novel corticosteroid Agamree and Italfarmaco’s Duvyzat are expanding the treatment options for DMD patients. Nevertheless, treatments that can meaningfully delay or halt the progressive muscle degeneration associated with the various forms of MD are still needed. The diverse landscape of therapies under development aims to fulfill the unmet needs for the treatment of DMD and other MD subtypes.

Questions answered

• What are the sizes of the key MD patient populations in the United States and EU5? How will they change in 2035?
• What is the expected uptake of key therapies for MD in 2025 and 2035?
• How is the gene therapy Elevidys being integrated into the current treatment landscape for ambulatory DMD patients?
• Will the safety issues reported in nonambulatory DMD patients impact clinical decisionmaking regarding Elevidys’s use in ambulatory patients?
• What are the greatest unmet clinical needs in the treatment of MD, according to experts? How well will pipeline therapies address these?
• Which emerging therapies will launch by 2035, and what will be their commercial impact on the MD market through 2035?

Content highlights

• Geography: United States, 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 2025 and 2035
• 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.