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 disease-modifying therapies (Sarepta’s Elevidys, Exondys 51, Vyondys 53, and Amondys 45; NS Pharma’s Viltepso; 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 in development for other forms of MD, including limb-girdle MD and Becker MD. Overall, the need for more-effective treatments for DMD and other forms of MD remains high.
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.
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.