Splice-switching targeted oligonucleotide drugs alter editing of gene transcript

Splice-switching targeted oligonucleotide drugs alter editing of gene transcript: "In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein. Developments in this rapidly advancing field have already led to promising treatments for such diseases as Duchenne Muscular Dystrophy and spinal muscular atrophy, as described in an article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website."



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Illinois Designates Week of June 23 as Duchenne Awareness Week! - PPMD Community

Illinois Designates Week of June 23 as Duchenne Awareness Week! - PPMD Community: "The State of Illinois is recognizing our 20th Annual Connect Conference in Chicago, Illinois, June 26-29, 2014 by declaring the week of June 23 as Duchenne Muscular Dystrophy Awareness Week! With over 500 families and Duchenne experts expected to attend from around the world, this will be our largest Annual Connect Conference to date."



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Webinar: Ataluren (Translarna) Update with PTC - PPMD Community

Webinar: Ataluren (Translarna) Update with PTC - PPMD Community: "In a webinar hosted by United Parent Project Muscular Dystrophy, Robert Spiegel, MD, FACP, chief medical officer of PTC Therapeutics, presented an overview of the positive opinion by the Committee for Medicinal Products for Human Use of the European Medicines Agency regarding the company’s application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy, and what this decision means for families living in and outside the EU."



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Summit Corp establishes US office | 17 June 2014 | Stock Market Wire

Summit Corp establishes US office | 17 June 2014 | Stock Market Wire: "Summit Corp has established US operations in Cambridge, Massachusetts and strengthened its drug development operations team following the recruitment of new clinical and preclinical employees.

The US operations are being formed to facilitate greater interactions with academic, clinical and business leaders in Summit's areas of therapeutic development.

Located at the new US office will be key members of the clinical development team: Dr Michael Boss, the overall leader of the utrophin modulation programme for DMD, and Dr Bindu Tejura who recently joined Summit as Vice President of Clinical Development and will be involved in both the DMD and CDI programmes."



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Clinical Development of HT-100 for Duchenne Has Resumed - PPMD Community

Clinical Development of HT-100 for Duchenne Has Resumed - PPMD Community: "Phase 1b/2a Clinical Program Resumes Enrollment and Dosing in DMD Patients 
Preliminary Clinical Data to be presented at Upcoming Medical Conference "



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» HT-100 Phase 1b/2a US clinical trial re-started in Duchenne Action Duchenne

» HT-100 Phase 1b/2a US clinical trial re-started in Duchenne Action Duchenne: "DART Therapeutics announced yesterday that it has resumed clinical development for its lead drug candidate, HT-100 (delayed-releasehalofuginone) an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. The company will continue to enroll and dose patients in this ongoing phase 1b/2a clinical study to evaluate the safety and tolerability of increasing doses of HT-100, and explore trends in a range of efficacy endpoints.

Please see the link for the press release for more information:"



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» HT-100 Phase 1b/2a US clinical trial re-started in Duchenne Action Duchenne

» HT-100 Phase 1b/2a US clinical trial re-started in Duchenne Action Duchenne: "DART Therapeutics announced yesterday that it has resumed clinical development for its lead drug candidate, HT-100 (delayed-releasehalofuginone) an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. The company will continue to enroll and dose patients in this ongoing phase 1b/2a clinical study to evaluate the safety and tolerability of increasing doses of HT-100, and explore trends in a range of efficacy endpoints.

Please see the link for the press release for more information:"



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