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 and Vyondys 53, 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 2029?
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 2029, and what will be their commercial impact on the MD market through 2029?
Raina Priyadarshini, Ph.D., is a senior analyst on the Infectious, Niche, and Rare Diseases team at Clarivate. Previously, she was an analyst in the biopharmaceutical industry, where she developed novel disease target-drug combinations for rare CNS and hematological disorders. Dr. Priyadarshini trained as a molecular biologist; her research focused on the interplay between tumor suppressor proteins and oncoproteins. She received her doctorate from the National Institute of Immunology.
Shilpa Thakur
Shilpa Thakur, M.P.H., is an epidemiologist at Clarivate. Previously, she monitored HIV sentinel surveillance in Himachal Pradesh. She has also studied the patterns of antimicrobial resistance in India. She received her M.P.H. from the Postgraduate Institute of Medical Education and Research with a specialization in epidemiology and biostatistics.