January 2017
Together we can Cure Duchenne!
Research
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Deflazacort Transition Program Reminder

Marathon submitted New Drug Applications (NDAs) to the Food and Drug Administration (FDA) last June for deflazacort. The NDAs have been accepted for filing and review. The FDA expects to complete its review of the applications in early February...

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BioMarin Transfers Duchenne Natural History Database to CureDuchenne To Improve Knowledge of Duchenne Patients and Improve Clinical Outcomes

CureDuchenne announced on January 10 that BioMarin Pharmaceutical Inc. has transferred its Duchenne natural history database to CureDuchenne, the venture philanthropy not-for-profit organization that is leading Duchenne research...

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Sarepta Therapeutics Enters into License Agreement with Nationwide Children’s Hospital for Galgt2 Gene Therapy Program

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial stage developer of innovative RNA-targeted therapeutics, announced on January 10 it has entered an exclusive license agreement with Nationwide Children’s Hospital, for their Galgt2 gene therapy program developed by researcher Dr. Paul Martin, Ph.D...

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Positive Preclinical Research on the Edasalonexent (CAT-1004) Program, a Potential Disease-Modifying Therapy for Duchenne Muscular Dystrophy, Published in JCI Insight

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced on January 4 the publication of preclinical data on the edasalonexent program, a potential disease-modifying therapy for Duchenne muscular dystrophy (DMD)...

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Eli Lilly and Company joins cTAP, helping to accelerate enriched clinical trial design for the provision of new treatments for Duchenne Muscular Dystrophy patients

Lilly to share data from Phase III trial of tadalafil in Duchenne, increasing knowledge of placebo-arm responses...

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Sarepta Therapeutics Announces EMA Validation of Eteplirsen Authorization Application for Treatment of Duchenne Muscular Dystrophy Amenable to Exon Skipping 51

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage developer of innovative RNA-targeted therapeutics, announced on December 19 that the European Medicines Agency (EMA) validated the previously submitted Marketing Authorization application (MAA) for eteplirsen to treat Duchenne muscular dystrophy amenable to exon 51 skipping...

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TRiNDS webinar

CureDuchenne hosted a webinar with TRiNDS, a specialized contract research organization (CRO) for neuromuscular diseases, on January 24.The webinar discussed TRiNDS new solution for the Duchenne research community...

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CureDuchenne Cares
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CureDuchenne Cares Improves Standard of Care and Clinical Trial Education Throughout the U.S. for Families Affected by Duchenne

It is vital that those caring for children and young adults with Duchenne muscular dystrophy have a thorough understanding of the disease and deliver appropriate care, based on the current research evidence...

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Equipment Options for Independent Lifestyles

Durable medical equipment helps individuals with Duchenne muscular dystrophy maintain independence throughout their lives. Watch this video from the CureDuchenne Cares Family Summit, featuring Kevin Philips, ATP/SMS, from Access Medical...

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Physical Therapist Darcy Peach Provides Peace of Mind to Duchenne Families

Darcy Peach discovered her calling to become a physical therapist while undergoing treatment for a volleyball injury in high school...

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

Third Annual Napa in Newport Set for March 4th with Napa Valley’s Finest Wines and Chef Amar Santana

We are only weeks away from the highly anticipated 3rd annual Napa in Newport Wine Auction, taking place at the Ritz Carlton Laguna Niguel on March 4th. More than 30 of Napa Valley’s finest vintners, along with wine enthusiasts and CureDuchenne supporters from around the country, will gather for Southern California’s premier wine event in saving the lives of children with Duchenne...

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

Biotech Leaders, Emerging Innovators, Investors and Patient Advocacy Group Representatives Participated in a Candid, Interactive and Enlightening Panel Discussion About the Evolving Landscape in Rare Disease Drug Discovery and Development

The panel, including Debra Miller, Founder of CureDuchenne; Henri Termeer, Former CEO; Board Member Genzyme; Aura Biosciences Alison Silva, President, Critical Outcome Technologies; and Philip J. Vickers, Ph.D., Global Head, R&D and Member of the Executive Committee Shire; and Flemming Ornskov, M.D., M.P.H., CEO of Shire, discussed how approaches to rare disease research, drug development, commercialization and investment have changed in recent years, and why and how they’ve chosen to pursue leadership and innovation in rare diseases...

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

Moody Foundation Provides Grant to Austin Gives Miles

Austin Gives Miles has once again been selected as a grant recipient from the Moody Foundation. The grant will match all donations raised by our 27 official charities through the Austin Gives Miles program, up to $10,000 per charity...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Profiled in Newport Beach Magazine

When their son, Hawken, was 4 years old, Paul and Debra Miller knew that something was wrong. They spent more than a year trying to get a diagnosis, and eventually found out that Hawken had Duchenne muscular dystrophy, a rare genetic disorder that causes progressive muscle deterioration and weakness...

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Upcoming Events
CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, January 28, 2017, Irving, TX

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on January 28 in Irving, Texas for family members and caregivers at the Residence Inn Dallas DFW Airport South/Irving - 2200 Valley View Ln, Irving, TX, 75062...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, February 25, 2017, Phoenix, AZ

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on February 25 in Phoenix, AZ for family members and caregivers at the Ray & Joan Kroc Crops Community Center, 1375 E. Broadway Rd., Phoenix, AZ, 85040...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Napa In Newport, March 4, 2017, Laguna, Nigel, CA

Reserve the date for Napa In Newport on March 4, 2017 at the Ritz-Carlton, Laguna Niguel. Napa In Newport brings together 30 of Napa’s most distinguished wineries to Orange County for an extraordinary wine and culinary experience...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Champions to CureDuchenne, April 8, 2017, Austin, TX

Save the date for the Champions to CureDuchenne gala on April 8 at the University of Texas Golf Club. Join us for a magical evening to find a cure for Duchenne muscular dystrophy. The event includes cocktails, dinner, casino, live and silent auction...

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