Therapeutic approaches for Duchenne muscular dystrophy: old and new

SJ Mackenzie, S Nicolau, AM Connolly… - Seminars in Pediatric …, 2021 - Elsevier
Duchenne muscular dystrophy (DMD) is marked by pathogenic variants in the DMD gene,
leading to reduced or absent dystrophin translation, muscle fiber destruction, loss of …

Comparing deflazacort and prednisone in Duchenne muscular dystrophy

WD Biggar, A Skalsky… - Journal of neuromuscular …, 2022 - content.iospress.com
Deflazacort and prednisone/prednisolone are the current standard of care for patients with
Duchenne muscular dystrophy (DMD) based on evidence that they improve muscle strength …

Evidence of polyphenols efficacy against dry eye disease

G Favero, E Moretti, K Krajčíková, V Tomečková… - Antioxidants, 2021 - mdpi.com
Dry eye disease is a multifactorial pathology compromising the quality of life of patients,
resulting in significant damage of the ocular surface and discomfort. The current …

Animal models for researching approaches to therapy of Duchenne muscular dystrophy

MI Zaynitdinova, AV Lavrov, SA Smirnikhina - Transgenic Research, 2021 - Springer
Duchenne muscular dystrophy (DMD) is a relatively widespread genetic disease which
develops as a result of a mutation in the gene DMD encoding dystrophin. In this review …

[HTML][HTML] Retinal dystrophins and the retinopathy of Duchenne muscular dystrophy

MTS Barboni, A Joachimsthaler, MJ Roux… - Progress in Retinal and …, 2023 - Elsevier
Duchenne muscular dystrophy (DMD) is caused by X-linked inherited or de novo DMD gene
mutations predominantly affecting males who develop early-onset muscle degeneration …

Functional and clinical outcomes associated with steroid treatment among non-ambulatory patients with Duchenne muscular dystrophy

CM McDonald, OH Mayer, KN Hor… - Journal of …, 2023 - journals.sagepub.com
Background: Evidence on the long-term efficacy of steroids in Duchenne muscular dystrophy
(DMD) after loss of ambulation is limited. Objective: Characterize and compare disease …

Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

CM McDonald, JR Marden, PB Shieh… - Journal of …, 2023 - becarispublishing.com
Aim: To examine benefits of corticosteroids for Duchenne muscular dystrophy (DMD) by age
and disease progression. Methods: Data from daily steroid users (placebo-treated) were …

CTLA4-Ig reduces muscle fiber damage in a model of Duchenne muscular dystrophy by attenuating pro-inflammatory gene expression in myeloid lineage cells.

M Wehling-Henricks, S Kok, H Gamboa… - The American Journal of …, 2025 - Elsevier
Duchenne muscular dystrophy (DMD) is a lethal, muscle-wasting, genetic disease that is
greatly amplified by an immune response to the diseased muscles. The mdx mouse model …

[HTML][HTML] Single-transcript multiplex in situ hybridisation reveals unique patterns of dystrophin isoform expression in the developing mammalian embryo

JCW Hildyard, AH Crawford, F Rawson… - Wellcome Open …, 2020 - ncbi.nlm.nih.gov
Background: The dystrophin gene has multiple isoforms: full-length dystrophin (dp427) is
principally known for its expression in skeletal and cardiac muscle, but is also expressed in …

Olive pomace phenolic compounds: From an agro-industrial by-product to a promising ocular surface protection for dry eye disease

N Katsinas, U Gehlsen, L García-Posadas… - Journal of Clinical …, 2022 - mdpi.com
Dry eye (DED) is a prevalent disease with immune-mediated inflammation as the principal
pathophysiological etiology. Olive pomace, the major by-product of the olive oil industry, is …