22 Apr
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DEPARTMENT OF DEFENSE DUCHENNE MUSCULAR DYSTROPHY RESEARCH PROGRAM FISCAL YEAR 2013 STRATEGIC PLAN

The Fiscal Year 2013 (FY13) Defense Appropriations Act provides for $3.2 million (M) to the Department of Defense Duchenne Muscular Dystrophy Research Program (DMDRP) to support innovative, high-impact Duchenne muscular dystrophy research. This program is administered by the US Army Medical Research and Materiel Command through the Office of the Congressionally Directed Medical Research Programs […]

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15 Apr
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Sarepta Therapeutics Announces FDA Will Consider Accelerated Approval for Eteplirsen After Further Review of Data on Dystrophin and Clinical Outcomes

Eteplirsen Manufacturing and Clinical Activities Continue as Planned CAMBRIDGE, MA–(Marketwired – April 15, 2013) – Sarepta Therapeutics, Inc. (NASDAQ: SRPT) today provided an update on its discussions with the U.S. Food and Drug Administration (FDA) regarding a potential application for accelerated approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). The FDA has […]

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11 Apr
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Drisapersen, Etiplersen, GSK, Prosensa and Sarepta, a Positive Week for Duchenne

Thanks to GSK, Prosensa and Sarepta and, the landscape is changing for the patients and families that are suffering from Duchenne, a severe muscle disease that shortens the life expectancy and quality of life for approximately 300,000 boys and young men worldwide. Even though these potential treatments that are currently in the news have not […]

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09 Apr
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DRISAPERSEN TREATMENT FOR DUCHENNE MUSCULAR DYSTROPHY: RESULTS OF A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED CLINICAL TRIAL

It’s been almost 10 years since CureDuchenne funded a little know biotech company, Prosensa, in the Netherlands.  With only $10,000 in our bank account, we committed to fund a $1.3 Million project to develop antisense (exon skipping) for Duchenne.  A few years later, venture capital firms validated our investment with several million dollars to continue their research.  […]

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08 Apr
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Update from GSK on drisapersen (previously GSK2402968)

GSK will be announcing results from their Phase llb study this week.  We will keep you updated on the news as it comes in.  Here is the note from GSK: ———————————————————————————————————————————————- To keep you updated on the progress of the drisapersen (previously GSK2402968) clinical development program, as you have requested, I am writing to inform […]

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05 Apr
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Sarepta Therapeutics Announces Eteplirsen Demonstrates Sustained Benefit on Walking Test Through 74 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy

Data to Be Presented at the Muscular Dystrophy Association Scientific Conference CAMBRIDGE, MA–(Marketwired – April 05, 2013) – Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced updated data from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results at 74 weeks […]

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21 Mar
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SUMMIT OUTLINES CLINICAL DEVELOPMENT PLANS FOR UTROPHIN MODULATOR PROGRAMME FOR DUCHENNE MUSCULAR DYSTROPHY

• First patient clinical trials of SMT C1100 expected to start H2 2013 •Summit to showcase programme at international scientific conference Oxford, UK, 21 March 2013 – Summit (AIM: SUMM), a drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy (‘DMD’) and C. difficile infections, today outlines its future plans for the continued […]

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04 Mar
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Drisapersen/GSK Update

Dear Debra, Since my last email you may have seen a recent story that appeared on an investment tips website regarding a presentation on drisapersen made at the Duchenne Parent Project Onlus meeting on 24th February 2013 in Rome, Italy. We appreciate that the story may raise questions for you, so I wanted to write and […]

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21 Feb
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FDA Webinar on Accelerated Approval Summary

    CureDuchenne was honored to host a webinar today, in collaboration with PPMD and MDA to discuss accelerated approval for drugs for rare diseases.   Dr. Robert Temple, director of the Office of Medical Policy of FDA’s Center for Drug Evaluation and Research presented detailed information on the different options available to speed the approval of […]

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29 Jan
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Prosensa Achieves Orphan Drug Status on its Entire

  Leiden, The Netherlands – 29 January 2013 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, announced it has achieved orphan drug designation in the EU and the US for all of its compounds for the treatment of Duchenne muscular dystrophy (DMD). The European Medicines Agency […]

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