Duchenne muscular dystrophy (DMD) affects the ensemble of the muscles of the organism: skeletal muscle, cardiac muscle, and some smooth muscle. The first symptoms appear in childhood at about 3 years of age. Only boys are affected. DMD is characterized by progressive muscle weakness of the limbs and the trunk. Going upstairs, then walking become difficult and then impossible. Respiratory and cardiac effects may be lethal.<\/p>\n
This program is based on the utilization of an AAV (Adeno-Associated Virus) vector carrying a transgene (U7) which permits exon skipping and the production of a \u201cquasi-dystrophin\u201d in the muscle of the patient. Locoregional administration by the intravenous route will allow treatment of the whole limb. The results of the preclinical studies in progress will be determinant for the trial in humans programmed for the end of 2013.<\/p>\n
For more information: the AAV-U7 gene therapy project<\/a><\/p>\n This open-label study for previously treated Ataluren (PTC124) patients with nonsense mutation dystrophinopathy aims at evaluating the long-term safety of ataluren, as determined by adverse events and laboratory abnormalities.<\/p>\n For further information: PTC124-GD-019-DMD project webpage<\/a><\/p>\n This phase III extension study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy aims at obtaining long term safety to ataluren as determined by adverse events and laboratory abnormalities. It also aims at assessing changes in physical function, pulmonary function and other important clinical and laboratory measures.<\/p>\n For further information: PTC124-GD-020e-DMD project webpage<\/a><\/p>\n This 2-part, randomized, double-blind, placebo-controlled, dose-titration, safety, tolerability and pharmacokinetics study (Part 1), followed by an open-label efficacy and safety evaluation (Part 2) of SRP-4053 in patients with Duchenne Muscular Dystrophy amenable to exon 53 skipping aims at assessing the safety, tolerability, efficacy and pharmacokinetics of SRP-4053.<\/p>\n For further information: SRP4053 project webpage<\/a><\/p>\n This phase IIb, open-label study aims at assessing the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of PRO045 in subjects with Duchenne Muscular Dystrophy.<\/p>\n For further information: Pro-045 project webpage<\/a><\/p>\n This phase I\/II, open-label, dose escalating with 48 weeks treatment study aims at assessing the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne Muscular Dystrophy.<\/p>\n For further information: Pro-053 project webpage\u00a0<\/a><\/p>\n This phase Ib, open-label study aims at evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy.<\/p>\n For further information: Rim4DMD project webpage<\/a><\/p>\n This phase IIIb, multi-centre, open-label extension study in male subjects with DMD who previously have been treated with drisapersen\u00a0aims\u00a0at assessing the long-term safety, tolerability and efficacy of drisapersen.<\/p>\n For further information: Drisapersen project webpage<\/a><\/p>\n This\u00a0natural history study aims at assessing\u00a0the sensitivity of neuromuscular functional characteristics following DMD natural history in order to select the\u00a0strongest follow-up characteristics during a therapy trial.<\/p>\n This natural history study aims at monitoring the clinical and radiological course of upper limb muscle impairment in patients with Duchenne Muscular Dystrophy, potentially treatable with AAV-mediated exon 53 skipping.<\/p>\n For further information: Pre U7-53 project webpage<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" Duchenne muscular dystrophy (DMD) affects the ensemble of the muscles of the organism: skeletal muscle, cardiac muscle, and some smooth muscle. The first symptoms appear in childhood at about 3 years of age. Only boys are affected. DMD is characterized by progressive muscle weakness of the limbs and the trunk. Going upstairs, then walking become … [Read more]<\/a><\/p>\n","protected":false},"author":3,"featured_media":0,"parent":4283,"menu_order":1,"comment_status":"closed","ping_status":"closed","template":"","meta":{"footnotes":""},"class_list":["post-4285","page","type-page","status-publish","hentry"],"_links":{"self":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages\/4285","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/comments?post=4285"}],"version-history":[{"count":3,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages\/4285\/revisions"}],"predecessor-version":[{"id":5717,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages\/4285\/revisions\/5717"}],"up":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages\/4283"}],"wp:attachment":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/media?parent=4285"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}Pharmacology trial of PTC124-GD-019-DMD<\/h2>\n
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Pharmacology trial of PTC124-GD-020e-DMD<\/h2>\n
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Pharmacology trial of SRP4053<\/h2>\n
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Pharmacology trial of Pro-045<\/h2>\n
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Pharmacology trial of Pro-053<\/h2>\n
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Pharmacology trial of Rim4DMD<\/h2>\n
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Pharmacology trial of BMN-051-302<\/h2>\n
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Longitudinal assessment of muscle damage in Duchenne Muscular Dystrophy\u00a0\u2014 NatHis-DMD<\/h2>\n
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Observational study of patients with Duchenne Muscular Dystrophy theoretically treatable with exon 53 skipping\u00a0\u2014 Pre U7-53<\/h2>\n
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