New Findings from Johns Hopkins University School of Medicine in the Area of Barth Syndrome Reported (AMCP Market Insights: Managed care considerations in Barth syndrome): Heart Disorders and Diseases – Barth Syndrome

2026 JUL 07 (NewsRx) -- By a News Reporter-Staff News Editor at Insurance Daily News -- New research on Heart Disorders and Diseases - Barth Syndrome is the subject of a report. According to news reporting originating from Baltimore, Maryland, by NewsRx correspondents, research stated, “Barth syndrome is an ultrarare, complex, multisystem, X-linked metabolic and neuromuscular disease, which poses significant and wide-ranging burden to patients and caregivers. Pharmacologic management focuses on treatment of disease manifestations and prevention of secondary complications.”

Our news editors obtained a quote from the research from the Johns Hopkins University School of Medicine, “Elamipretide, the first treatment indicated specifically for improving muscle strength in Barth syndrome, was approved in 2025 via accelerated approval warranting guidance for payers. To discuss managed care considerations in Barth syndrome including management of elamipretide, AMCP Market Insights virtually convened an expert panel of managed care stakeholders in March 2026. This article provides a qualitative summary of the panel discussion along with key insights and suggested payer practices meant to support informed coverage decisions and guide future work such as collaboration, research, and advocacy. Key insights highlight that there are unique challenges in generating clinical trial evidence for treatments in ultrarare conditions, which leads to difficulties determining the value of these treatments and differences in whether they are covered among payers. Additionally, there are numerous elements of care to which patients with Barth syndrome and their caregivers need equitable access, which is complicated by involving multiple specialists and fragmentation.”

According to the news editors, the research concluded: “Suggested payer practices involve education, care delivery, and coverage and benefit design.”

This research has been peer-reviewed.

For more information on this research see: AMCP Market Insights: Managed care considerations in Barth syndrome. Journal of Managed Care & Specialty Pharmacy, 2026;32. Journal of Managed Care & Specialty Pharmacy can be contacted at: Acad Managed Care Pharmacy, 100 N Pitt St, 400, Alexandria, VA 22314-3134, USA.

The news editors report that additional information may be obtained by contacting Hilary J. Vernon, Johns Hopkins University School of Medicine, Baltimore, MD. Additional authors for this research include Bridget Flavin, W. Todd Cade, Zachary Contreras, Michael Kobernick, Farzana Rahman, Terry Richardson and Denise Wolff.

Publisher contact information for the Journal of Managed Care & Specialty Pharmacy is: Acad Managed Care Pharmacy, 100 N Pitt St, 400, Alexandria, VA 22314-3134, USA.

(Our reports deliver fact-based news of research and discoveries from around the world.)