Joana P. Domingos

Great Ormond Street Institute Of Child Health Biomedical Research Centre
London, ENG, United Kingdom

Last Updated: 09/26/2026

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Found 15 publications

T Cell Responses to Dystrophin in a Natural History Study of Duchenne Muscular Dystrophy.

T Cell Responses to Dystrophin in a Natural History Study of Duchenne Muscular Dystrophy.

Journal: Human gene therapy
Published: December 01, 2022

Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy.

Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy.

Journal: PloS one
Published: June 20, 2022

High-Throughput Digital Image Analysis Reveals Distinct Patterns of Dystrophin Expression in Dystrophinopathy Patients.

High-Throughput Digital Image Analysis Reveals Distinct Patterns of Dystrophin Expression in Dystrophinopathy Patients.

Journal: Journal of neuropathology and experimental neurology
Published: September 09, 2021

Genotype-related respiratory progression in Duchenne muscular dystrophy-A multicenter international study.

Genotype-related respiratory progression in Duchenne muscular dystrophy-A multicenter international study.

Journal: Muscle & nerve
Published: April 28, 2021

Novel free-circulating and extracellular vesicle-derived miRNAs dysregulated in Duchenne muscular dystrophy.

Novel free-circulating and extracellular vesicle-derived miRNAs dysregulated in Duchenne muscular dystrophy.

Journal: Epigenomics
Published: November 20, 2020

Blood-derived biomarkers correlate with clinical progression in Duchenne muscular dystrophy.

Blood-derived biomarkers correlate with clinical progression in Duchenne muscular dystrophy.

Journal: Journal of neuromuscular diseases
Published: May 12, 2020

Rimeporide as a first- in-class NHE-1 inhibitor: Results of a phase Ib trial in young patients with Duchenne Muscular Dystrophy.

Rimeporide as a first- in-class NHE-1 inhibitor: Results of a phase Ib trial in young patients with Duchenne Muscular Dystrophy.

Journal: Pharmacological research
Published: March 20, 2020

Cardiorespiratory Progression Over 5 Years and Role of Corticosteroids in Duchenne Muscular Dystrophy: A Single-Site Retrospective Longitudinal Study.

Cardiorespiratory Progression Over 5 Years and Role of Corticosteroids in Duchenne Muscular Dystrophy: A Single-Site Retrospective Longitudinal Study.

Journal: Chest
Published: December 02, 2019

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy.

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy.

Journal: Neurology
Published: June 11, 2019

Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy.

Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy.

Journal: PloS one
Published: January 28, 2019

Downregulation of miRNA-29, -23 and -21 in urine of Duchenne muscular dystrophy patients.

Downregulation of miRNA-29, -23 and -21 in urine of Duchenne muscular dystrophy patients.

Journal: Epigenomics
Published: March 23, 2018

Outcome measures in Duchenne muscular dystrophy: sensitivity to change, clinical meaningfulness, and implications for clinical trials.

Outcome measures in Duchenne muscular dystrophy: sensitivity to change, clinical meaningfulness, and implications for clinical trials.

Journal: Developmental medicine and child neurology
Published: November 28, 2017
Showing 1-12 of 15

Last Updated: 09/26/2026