Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophy

• Published in: Cellular and molecular life sciences : CMLS Vol. 70; no. 23; pp. 4585 - 4602
• Main Authors: Govoni, Alessandra, Magri, Francesca, Brajkovic, Simona, Zanetta, Chiara, Faravelli, Irene, Corti, Stefania, Bresolin, Nereo, Comi, Giacomo P
• Format: Journal Article
• Language: English
• Published: Basel Springer-Verlag 01.12.2013; Springer Basel; Springer Nature B.V
• Subjects: Animals; Becker's muscular dystrophy; Biochemistry; Biomarkers - blood; Biomedical and Life Sciences; Biomedicine; Cell Biology; Cell therapy; Clinical outcomes; Clinical trials; Clinical Trials as Topic - methods; Degeneration; Drug Therapy - methods; Duchenne's muscular dystrophy; Dystrophy; Exon skipping; exons; experimental design; Gene therapy; Genetic disorders; Humans; Immunomodulation; Life Sciences; muscle tissues; Muscular dystrophy; Muscular Dystrophy, Duchenne - blood; Muscular Dystrophy, Duchenne - physiopathology; Muscular Dystrophy, Duchenne - therapy; Outcome Assessment, Health Care - methods; Reproducibility of Results; Review; Stem Cell Transplantation - methods; tissue degeneration; Walking - physiology; Outcome measures; Clinical trial; Readthrough; Duchenne muscular dystrophy; Exon skipping; New therapeutic approaches

Muscular dystrophy is a heterogeneous group of genetic disorders characterised by progressive muscle tissue degeneration. No effective treatment has been discovered for these diseases. Preclinical and clinical studies aimed at the development of new therapeutic approaches have been carried out, prim...