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?

Table of contents

  • Muscular Dystrophy - Landscape & Forecast - Disease Landscape & Forecast

Author(s): Nuno Tiago Giao Antunes, PhD; Shilpa Thakur

Nuno T. Antunes, Ph.D., is senior business insights analyst on the Infectious, Niche, and Rare Diseases team at Decision Resources Group, specializing in antibacterial agents. Previously, he was a Latin America Market Access senior analyst in the Global Market Access Insights Team, where he developed expertise in market access, pricing and reimbursement, health technology assessment, and health policy.

Nuno holds a Ph.D. in animal health from the Universidad de las Palmas de Gran Canaria, Spain, and a D.V.M. degree from the Universidade de Trás-os-Montes e Alto Douro, Portugal. Prior to joining DRG, he conducted research in antimicrobial resistance and antimicrobial development, and worked in the medical devices industry as a scientist.

Shilpa Thakur is a medical graduate with a M.P.H from the Postgraduate Institute of Medical Education and Research with a specialization in epidemiology and biostatistics. She specializes in developing epidemiological forecasts for the multiple indications within the DRG syndicated portfolio.

Prior to joining Decision Resources, she monitored HIV sentinel surveillance 2016-2017 in Himachal Pradesh. She also has worked on to see the patterns of Antimicrobial resistance in India.