Muscular Dystrophy | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2021

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 MD is glucocorticoids, which have proven effective in delaying the loss of ambulation. Recent conditional approvals of genotype-specific, disease-modifying therapies such as Sarepta’s Exondys 51, Vyondys 53, and Amondys 45; Nippon Shinyaku’s Viltolarsen; and PTC Therapeutics’ Translarna have expanded treatment options for DMD patients, but there are lingering concerns about these drugs’ clinical efficacy. Additionally, many therapies with diverse mechanisms of action are being developed to treat other forms of MD, including limb-girdle MD and Becker MD. Overall, there is still high unmet need for more-effective treatments for DMD and other forms of MD.

QUESTIONS ANSWERED

• What are the sizes of key MD patient populations in the United States and EU5 countries, and how will the population change through 2031?
• What is the current treatment landscape in MD, and where are the greatest unmet clinical needs according to experts?
• How well will pipeline therapies address current treatment gaps?
• Which emerging therapies will launch before 2031, and what will be their commercial impact on the MD market through 2031?

GEOGRAPHIES

United States, 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 Becker MD, limb-girdle MD, facioscapulohumeral MD, and myotonic dystrophy.

FORECAST

Drug-level sales and patient shares of key muscular dystrophy therapies through 2031

EMERGING THERAPIES

Phase III/PR: 6 drugs; Phase I/II: > 10 drugs; coverage of select early-phase and preclinical products.

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.