Muscular Dystrophy | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2020

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. There is a high unmet need for effective treatments that can meaningfully delay or halt progressive muscle degeneration in Duchenne muscular dystrophy (DMD), the most common childhood-onset form, as well as in other forms of MD. The standard treatment option for MD patients is glucocorticoids, which have proven effective in delaying 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; however, there are lingering concerns about the clinical efficacy of these drugs. 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 additional and, ideally, more-effective medications 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 2030?
• What are the current treatment landscape and medical practice 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 2030, and what will be their commercial impact on the MD market through 2030?