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Browse selected scientific articles published in journals across the fields of molecular genetics, metabolism, and neurology to facilitate your understanding of the spectrum, molecular and genetic basis, and management of Duchenne muscular dystrophy (DMD)

Please select a publication review of interest below:

Duchenne muscular dystrophy (DMD)

Publication: Transition of patients with Duchenne muscular dystrophy from paediatric to adult care: An international Delphi consensus study

This article, in the European Journal of Paediatric Neurology, summarizes the Delphi consensus study, which aims to establish best practices for the transition of young people with Duchenne muscular dystrophy (DMD) to adult care, and ensure continuity of treatment

Duchenne muscular dystrophy (DMD)

Publication: Prednisone and deflazacort in Duchenne muscular dystrophy: A patient perspective and plain language summary publication of the Cincinnati study

This article in the Journal of Comparative Effectiveness Research summarizes the Cincinnati study, which investigated the effectiveness of 2 corticosteroid treatments in prolonging motor function in Duchenne muscular dystrophy (DMD)

Duchenne muscular dystrophy (DMD)

Publication: Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: Experience at a single, large care center

This research article, published in the Journal of Comparative Effectiveness Research, provides results from a retrospective real-world analysis of corticosteroid use in patients with Duchenne muscular dystrophy (DMD)

Duchenne muscular dystrophy (DMD)

Publication: Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy

This research article, published in the Journal of Comparative Effectiveness Research, provides results of meta-analyses comparing the efficacy of different corticosteroids in the management of nonsense mutation Duchenne muscular dystrophy (nmDMD)

Duchenne muscular dystrophy (DMD)

Publication: Cost of illness in patients with Duchenne muscular dystrophy in Portugal: The COIDUCH study

This article, published in PharmacoEconomics, provides results from an investigation to determine the cost of illness of Duchenne muscular dystrophy (DMD)

Duchenne muscular dystrophy (DMD)

Publication: Metabolite V, an epoxide species, is a minor circulating metabolite in humans following a single oral dose of deflazacort

This research article, published in Pharmacology Research & Perspectives, investigates the plasma pharmacokinetics, metabolite profiles and their quantitative exposures in humans following a single oral dose of deflazacort

Duchenne muscular dystrophy (DMD)

Publication: In vitro cytochrome P450- and transporter-mediated drug interaction potential of 6β-hydroxy-21-desacetyl deflazacort: A major human metabolite of deflazacort

This research article, published in Pharmacology Research & Perspectives, evaluates cytochrome P450 (CYP)- and transporter-mediated drug interaction potentials of 6β-hydroxy-21-desacetyl deflazacort (6β-OH-21-desDFZ)

Learn more about our phase I–III clinical trials, patient registries and natural history studies in DMD.

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MED-US-CORP-2500008 | November 2025

MED-US-DMD-2600003 | February 2026

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