Sage Journals: Discover world-class research

Open access

Case report

First published 2018

One-year follow up of three Italian patients with Duchenne muscular dystrophy treated with ataluren: is earlier better?

Lucia Ruggiero, Rosa Iodice, Marcello Esposito [...]

Therapeutic Advances in Neurological Disorders

Open access

Research article

First published 2024-12

Safety and effectiveness of ataluren in patients with Duchenne muscular dystrophy: single-center experience from Saudi Arabia

Mushtaha Ahmad, Alaa ElRasoul, Raneem Sedayou [...]

Journal of International Medical Research

Open access

Research article

First published 2023-11

Dihydroartemisinin and zerumbone esters of ataluren and its analogs as anticancer agents and EGFR inhibitors

Duc Quan Tran, Ngoc Hung Truong, Thi Hoang Anh Nguyen [...]

Journal of Chemical Research

Retricted access

Other

First published 2010-9

Read-Through Strategies for Suppression of Nonsense Mutations in Duchenne/ Becker Muscular Dystrophy: Aminoglycosides and Ataluren (PTC124)

Richard S. Finkel

Journal of Child Neurology

Retricted access

Review article

First published 2012-7

Cystic Fibrosis Transmembrane Conductance Regulator–Modifying Medications: The Future of Cystic Fibrosis Treatment

Rebecca S Pettit

Annals of Pharmacotherapy

Retricted access

Research article

First published 2021-03

Whole-Exome Sequencing Identifies Small Mutations in Pakistani Muscular Dystrophy Patients

Mehwish Zehravi, Mohsin Wahid, Junaid Ashraf [...]

Genetic Testing and Molecular Biomarkers

Open access

Review article

First published 2023-12

Targeted Molecular Strategies for Genetic Neurodevelopmental Disorders: Emerging Lessons from Dravet Syndrome

Robert Lersch, Rawan Jannadi, Leonie Grosse [...]

The Neuroscientist

Open access

Research article

First published 2020-7

Targeting translational read-through of premature termination mutations in BMPR2 with PTC124 for pulmonary arterial hypertension

Lu Long, Xudong Yang, Mark Southwood [...]

Pulmonary Circulation

Open access

Review article

First published 2022

Timing of Clinical Interventions in Patients With Duchenne Muscular Dystrophy: A Systematic Review and Grading of Evidence

E. Landfeldt, N. Ferizović, K. Buesch

Journal of Neuromuscular Diseases

Open access

Review article

First published 2024-01-02

Factors Associated with Respiratory Health and Function in Duchenne Muscular Dystrophy: A Systematic Review and Evidence Grading

E. Landfeldt, A. Aleman, S. Abner [...]

Journal of Neuromuscular Diseases

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