November 2015
Together we can Cure Duchenne!
Research
FDA Advisory Committee Discusses Clinical Data Package for BioMarin's Kyndrisa(TM) (drisapersen) for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

FDA Advisory Committee Discusses Clinical Data Package for BioMarin's Kyndrisa(TM) (drisapersen) for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced on November 24 that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) met to discuss the data submitted to support the New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping...

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Sarepta Therapeutics Announces Publication of Positive Long-Term Safety and Efficacy Data for Eteplirsen in the Annals of Neurology

Sarepta Therapeutics Announces Publication of Positive Long-Term Safety and Efficacy Data for Eteplirsen in the Annals of Neurology

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-based therapeutics, announced on November 17 that the Annals of Neurology published online positive efficacy and safety results from a Phase IIb long-term open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping...

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BioMarin Announces Data Analysis Demonstrating Consistent Efficacy of Kyndrisa™ (drisapersen) in Comparable Patients Across Three Randomized Studies

BioMarin Announces Data Analysis Demonstrating Consistent Efficacy of Kyndrisa™ (drisapersen) in Comparable Patients Across Three Randomized Studies

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced on November 20 data analysis for Kyndrisa demonstrating consistent evidence of efficacy in comparable patients across three randomized, placebo-controlled studies that were conducted contemporaneously...

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Duchenne Research Highlights from MDA Conference

Summit Therapeutics and the University of Oxford Announce Multi-Year Extension of Strategic Alliance for Development of Utrophin Modulators for DMD

Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ("DMD") and Clostridium difficile infection, announces a multi-year extension of its exclusive strategic alliance with the University of Oxford until November 2019, with an option to extend it for a further 12 months, to support and accelerate development of future generation utrophin modulators for the treatment of the progressive muscle wasting disorder, DMD...

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Duchenne muscular dystrophy is a stem cell disease

A new study from The Ottawa Hospital and the University of Ottawa is poised to completely change our understanding of Duchenne muscular dystrophy and pave the way for far more effective treatments...

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Target gene identified for therapies to combat muscular dystrophy

Researchers at the University of São Paulo's Bioscience Institute (IB-USP) in Brazil have shown that a gene called Jagged1, or JAG1 for short, could be a target for the development of new approaches to treat Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration...

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Carmeseal-MD™ Protects Skeletal Limb Muscle, the Third Major Target Muscle of Therapeutic Relevance in Duchenne Muscular Dystrophy (DMD)

Phrixus Pharmaceuticals welcomes the results from a preclinical study that examines the effects of Carmeseal-MD (P-188 NF) in the mdx mouse, a leading preclinical model of DMD...

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Santhera Reports New Data and Updates on Regulatory Filings for Raxone® (idebenone) in Duchenne Muscular Dystrophy (DMD)

Santhera Pharmaceuticals (SIX: SANN) announces that it has now completed comparative analyses of the respiratory outcomes for patients in its successful Phase III DELOS trial with data from a natural history DMD patient cohort collected by the Cooperative International Neuromuscular Research Group (CINRG)...

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Cardiac Trial for Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy

Did you know that heart failure is very common and often fatal in patients with Duchenne muscular dystrophy...

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Study Identifies Protein Biomarkers for Duchenne Muscular Dystrophy

Using a large-scale technological approach, a team of researchers has uncovered several dozen proteins that could serve as biomarkers for the muscle wasting disease, Duchenne muscular dystrophy (DMD)...

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You are Pioneers Once Again

We are pleased to share the following letter from the CEO of PTC Therapeutics to the Duchenne community. CureDuchenne funded PTC Therapeutics back in 2003 and we are proud to have helped advance the development of Translarna...

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BioMarin: Quarterly Update to the Duchenne Community

BioMarin: Quarterly Update to the Duchenne Community

Welcome to the second of the BioMarin quarterly updates. As you can imagine, it has been an incredibly busy time at BioMarin. Drisapersen, our lead exon skipping compound to treat Duchenne MuscularDystrophy (DMD) amenable to exon 51 skipping, is currently under review by the US Food & Drug Administration (FDA) and the European Medicines Agency (EMA)...

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CureDuchenne Cares
Abilities Expo Video Blog

Exciting Developments Coming in 2016 to CureDuchenne Cares

With a new year comes new possibilities! This statement couldn't be truer than with CureDuchenne Cares, the organization's interactive education and outreach program designed to increase the health and wellbeing of those living with Duchenne muscular dystrophy...

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Abilities Expo Video Blog

Presentation of Redefining the Standard of Care for Duchenne Muscular Dystrophy Now Available

CureDuchenne is a proud to have partnered with Medical Learning Institute, Inc., and PeerView Institute for Medical Education on a Duchenne symposium for child neurologists...

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Abilities Expo Video Blog

Transfer Techniques to Protect Duchenne Patients and Caregivers

Moving someone who has Duchenne from one location to another is no easy task, especially when considering patient and caregiver safety...

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News
Vince Young Hosts Champions to CureDuchenne Event to Find a Cure for Duchenne Muscular Dystrophy

Tyler Armstrong Challenges You to Pick Your Peak and Climb to CureDuchenne

Tyler Armstrong, the 11-year-old mountaineering phenomenon, continues his quest to Climb to CureDuchenne. Tyler has been training and helping to raise awareness and money to help find a cure for Duchenne muscular dystrophy...

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Vince Young Hosts Champions to CureDuchenne Event to Find a Cure for Duchenne Muscular Dystrophy

Give a Percent of Your Amazon Purchase to CureDuchenne

Give a percent of your Amazon purchase to CureDuchenne this holiday season by making your purchases on Amazon Smile. Amazon Smile donates 0.5% of all qualifying purchases to the charity chosen by buyer...

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Recent Success
CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne’s Debra Miller and Jak Knowles Spoke on Venture Philanthropy and Duchenne at Recent Conferences

CureDuchenne’s success in venture philanthropy has attracted the attention of the medical research community around the world. Last week, CureDuchenne CEO Debra Miller and Vice President of CureDuchenne Medical and Scientific Affairs Jak Knowles spoke at two different conferences about venture philanthropy and how it positively influences otherwise unapproachable biomedical technologies...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

New Videos from Getzlaf Golf

This year’s Getzlaf Golf Shootout was a tremendous success. Check out these two new event videos...

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Upcoming Events
CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, January 30, Austin, Texas

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on January 30 in Austin, Texas for family members and caregivers at the Thompson Center: 2405 Robert Dedman Drive, Austin, TX 78712...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, February 20, Riverside, California

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on February 20 in Riverside, California for family members and caregivers at the Courtyard Riverside: 1510 University Avenue, Riverside, CA 92507...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Napa in Newport, February 27, 2016, St. Regis Hotel, Dana Point, CA

Napa in Newport is Southern California's premier wine event. Napa in Newport brings together as many as 40 of Napa's best wineries and world class chef for a truly unforgettable experience...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Champions to CureDuchenne Austin, April 9, 2016, Austin, TX

Save the date for the Champions to CureDuchenne Austin event on April 9, 2016 in Austin, TX. This annual event will benefit CureDuchenne and is presented by Johnny Carino’s...

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