Saturday 24 May 2014

» Prosensa update on regulatory discussions and ongoing plans for re-dosing Action Duchenne

» Prosensa update on regulatory discussions and ongoing plans for re-dosing Action Duchenne: "Prosensa today provided an update to the patient-led organisations. They have been encouraged in their discussions with the regulatory authorities and are on-track for a pathway forward by the end of June.

The team also informed us that they are also on-track for re-dosing in the first group of boys in the third quarter of this year. Re-dosing is taking a staged approach, under new or existing treatment protocols or via expanded access programs. In a first wave, they will initiate re-dosing at sites in both US and Europe and as stated previously are actively working with these sites and investigators to execute these plans."



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EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna: "PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the company's application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older."



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Thursday 22 May 2014

Summit updates on Phase 1b trial for SMTC1100 | 21 May 2014 | Stock Market Wire

Summit updates on Phase 1b trial for SMTC1100 | 21 May 2014 | Stock Market Wire: "StockMarketWire.com - Summit Corporation says SMT C1100 for the treatment of Duchenne Muscular Dystrophy (DMD) has successfully met its primary endpoint of safety and tolerability in a Phase 1b clinical trial in patients with the disease.

SMT C1100 is an oral small molecule utrophin modulator that has the potential to treat all patients with DMD, regardless of the underlying dystrophin fault causing the disease.

The Phase 1b dose-escalating trial was conducted in 12 patients with DMD aged between 5 and 11 years old.

"These preliminary results show that SMT C1100 was safe and well tolerated at all doses tested in the study, and that there were no issues with patient compliance," Summit said. "



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Tuesday 13 May 2014

20th Annual Connect Conference - Parent Project Muscular Dystrophy

20th Annual Connect Conference - Parent Project Muscular Dystrophy: "This year marks PPMD's 20th Annual Connect Conference in Chicago, June 26-29, 2014. What started as a few dozen parents and a handful of experts gathered to discuss and learn all they could about Duchenne, has grown into a meeting where families, physicians, researchers, industry leaders, and experts of all kinds gather to speak face-to-face about Duchenne."



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Unprecedented Collection of Duchenne Experts Developing Guidance for FDA - PPMD Community

Unprecedented Collection of Duchenne Experts Developing Guidance for FDA - PPMD Community: "The process for writing a Draft Guidance on Duchenne for the FDA and industry is in full swing.
As you know, the purpose of this guidance is to assist sponsors in the clinical development of medical products (i.e., human drugs and therapeutic biological products) for the treatment of Duchenne over the entire spectrum of the disease (ambulatory and non ambulatory). It is the result of the first collaboration between the FDA and a rare disease community to produce clinical guidance in their respective disease area."



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Results of PPMD-Funded Study Prompt Phase III Study of Tadalafil in Duchenne - PPMD Community

Results of PPMD-Funded Study Prompt Phase III Study of Tadalafil in Duchenne - PPMD Community: "A recent PPMD-funded study at Cedars Sinai Medical Center in Los Angeles (published in the May 7, 2014, online issue of Neurology®) demonstrates that those with Duchenne have impaired circulation in their muscles, which may contribute to muscle weakness, and that the approved vasodilator tadalafil can restore that circulation to normal levels. "



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Friday 9 May 2014

DuchenneConnect Annual Report: Registry Growth & Expanded Services - PPMD Community

DuchenneConnect Annual Report: Registry Growth & Expanded Services - PPMD Community: "This past year has brought many new and exciting changes to DuchenneConnect, the robust and cutting-edge registry and resource that connects Duchenne and Becker patients with actively recruiting clinical trials and research studies, and educates patients and families about Duchenne and Becker research. The registry, which was established in 2007, has matured beyond a standard registry and into an innovative resource for the entire Duchenne and Becker community. This 2013 Annual Report (PDF) highlights how we have grown and expanded our services over the past year. "



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