Top 5 Duchenne Muscular Dystrophy Content of 2024

This year’s top stories on Duchenne muscular dystrophy (DMD) managed care included a novel imaging method for tracking disease progression, an FDA approval of the first nonsteroidal therapy, and the benefits of cycling training for mobility. Experts also highlighted the need for better patient-reported outcomes and explored the insurance value of novel treatments for rare diseases like DMD.

Here are AJMC.com’s 5 most-viewed DMD pieces of 2024. You can check out all our DMD coverage here.

5. Novel Imaging Algorithm Shows Promise in DMD Evaluation

A novel robust reference frequency method (RRFM) outperformed traditional imaging techniques in assessing ambulatory function progression in patients with DMD. Researchers found that RRFM-based ultrasound attenuation imaging significantly reduced measurement bias and offered superior diagnostic performance compared with other imaging methods, such as Nakagami and entropy analysis. With an area under the receiver operating characteristic curve of 0.92 in differentiating early and late ambulatory stages, RRFM showed potential as a noninvasive, reliable tool for managing DMD care.

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4. Health State Utility Scores for DMD-Related Impairment Need to Better Reflect Patient Input

Research at the beginning of the year highlighted the need for patient-reported outcomes to better capture how DMD affects patients’ quality of life. Using tools like the Health Utilities Index and EQ-5D-5L, researchers found that health utility scores declined as disease severity increased, with nonambulatory patients reporting the lowest scores. The findings emphasize the unique insights patients can provide, which may not always align with caregiver assessments.

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3. Cycling Training Shows Protective Effects on Gait, Balance in DMD

Cycling training demonstrated benefits for maintaining and improving balance and gait in children with DMD, outperforming traditional home-based exercise in enhancing antero-posterior balance. Both exercise approaches preserved key mobility parameters, which are vital for maintaining quality of life in this progressive condition. While the study was limited by its short duration and lack of follow-up, it highlights the potential of cycling as a targeted rehabilitation strategy for patients with DMD.

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2. Givinostat Receives Full FDA Approval for Duchenne Muscular Dystrophy

In March, the FDA approved givinostat (Duvyzat) for boys aged 6 years and older with DMD, marking it as the first nonsteroidal therapy for the condition. A histone deacetylase inhibitor, givinostat has shown in clinical trials to improve functional outcomes like stair-climbing ability and delay fat infiltration in muscles when combined with corticosteroids. The approval followed positive results from the EPIDYS trial, which demonstrated reduced disease progression and manageable side effects.

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1. Insurance Insights: Dr Jason Shafrin Estimates DMD Insurance Value

Jason Shafrin, PhD, joined an episode of Managed Care Cast to discuss his study published in The American Journal of Managed Care^®, which estimated the insurance value of novel treatments for DMD. Using survey data from individuals planning to have children, the study highlights how innovative therapies for rare diseases like DMD impact not only health outcomes but also financial risk for families. On the podcast, Shafrin underscored the importance of factoring insurance value into health economic analyses, particularly for diseases with significant unmet needs.

Listen to the podcast.