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.
United States, EU5
Six country-specific interviews with thought-leading neurologists
Supported by survey data collected for this study
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.
Drug-level sales and patient shares of key muscular dystrophy therapies through 2031
Phase III/PR: 6 drugs; Phase I/II: > 10 drugs; coverage of select early-phase and preclinical products.
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.