July 2015
Together we can Cure Duchenne!
Research
PTC Announces Translarna™ Access Program In Duchenne Muscular Dystrophy for Siblings of Patients Participating in PTC Clinical Trials

BioMarin Announces FDA Accepts Drisapersen NDA for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced on June 29 the U.S. Food and Drug Administration (FDA) has accepted for review the submission of a New Drug Application (NDA) for drisapersen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, and the Prescription Drug User Fee Act (PDUFA) goal date for a decision is December 27, 2015...

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Duchenne Research Highlights from MDA Conference

Sarepta Therapeutics Completes NDA Submission to FDA for Eteplirsen for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, announced the completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for eteplirsen on June 26, 2015...

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PTC Announces Translarna™ Access Program In Duchenne Muscular Dystrophy for Siblings of Patients Participating in PTC Clinical Trials

CureDuchenne’s Funding Bears Fruit with Drisapersen: First-Ever New Drug Application (NDA) Accepted by Food and Drug Administration (FDA) For Duchenne Muscular Dystrophy

CureDuchenne applauded the U.S. Food and Drug Administration (FDA), which took the critical step of accepting the first-ever New Drug Application (NDA) for a possible treatment for those who suffer with Duchenne muscular dystrophy. The FDA said it will accept BioMarin Pharmaceutical’s NDA for drisapersen, the first Duchenne drug ever to reach this stage...

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Duchenne Research Highlights from MDA Conference

BioMarin Announces EMA Validates MAA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin Pharmaceutical Inc. (BMRN) announced June 25 the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy amenable to exon 51 skipping...

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PTC Announces Translarna™ Access Program In Duchenne Muscular Dystrophy for Siblings of Patients Participating in PTC Clinical Trials

SKIP-NMD clinical trial now progressing to part 2

The SKIP-NMD team successfully completed the first part of the clinical trial, a dose escalation phase with no significant problem for the 12 children recruited so far, and recently obtained the go-ahead from the data safety monitoring board to initiate the second, maintenance phase of the study...

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CureDuchenne Cares
Abilities Expo Video Blog

CureDuchenne Cares Launches Website to Share Valuable Resources with the Duchenne Community

CureDuchenne unveiled the CureDuchenne Cares website on July 15 to provide families with vital health information focused on managing the devastating effects of the disease...

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Abilities Expo Video Blog

New CureDuchenne Cares Video

Check out the new CureDuchenne Cares video...

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Abilities Expo Video Blog

Parents Learn Valuable Information at CureDuchenne Cares Workshops

Parents, caregivers and physical therapists are gaining hands on knowledge about Duchenne care, stretching and the status of clinical trials and research at CureDuchenne Cares workshops...

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Abilities Expo Video Blog

Video Blog 14 - CureDuchenne Cares Answered The Common Stretching Questions

CureDuchenne Cares programs are held across the country. Many questions are asked and answered at each session...

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

Determined to Defeat Duchenne

Debra Miller is featured in an article in the Orange County Business Journal about finding a cure for Duchenne. The article highlights CureDuchenne’s venture philanthropy model and CureDuchenne’s role in funding critical Duchenne research...

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

Ryan Getzlaf to Host the 5th Annual Getzlaf Golf Shootout on September 12 and 13

Anaheim Ducks captain Ryan Getzlaf and his wife Paige are hosting the fifth annual Getzlaf Golf Shootout to benefit CureDuchenne on September 12 and 13. The party starts Saturday night when Ryan and Paige roll out the red carpet at Sutra Lounge in Costa Mesa for the pre-golf reception...

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

Beer Enthusiasts Raise Money for CureDuchenne

More than 2,500 beer enthusiasts joined together for a celebration of beer, Knoxville style at the 5th Annual Knoxville Brewfest on June 20 to benefit CureDuchenne. The fest raised more than $35,000 to help fund research to 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

Jim Ruetz Completes the Principalities of Europe Motorcycle Ride to Find a Cure for Duchenne

Jim Ruetz, a business man from Oak Creek, Wisconsin, completed The Principalities of Europe Motorcycle Ride for CureDuchenne on July 7, 2015. Over the 24 day trip, Ruetz traveled 2,307 miles by road, visited 13 countries, and traversed 3 mountain ranges...

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Upcoming Events
CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Continuing Education Course for Physical Therapists, August 14, 2015, Shakopee, MN

This course is ideal for physical therapists, occupational therapists, adapted physical educators and would be beneficial for others involved in the care of persons with Duchenne such as speech and language pathologists, educators, physicians, and nursing professionals...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Duchenne Families, August 15, 2015, Bloomington, MN

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on August 15 in Bloomington, MN. The session provides updates on stretching, equipment, activity and school modification, mobility management, research and clinical trials...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Climb to CureDuchenne, August 27-31, Colorado

From August 27-31, Ryan Christiansen and a team of hikers called the Darkside Warriors will be doing a double summit on Grays Peak and Torreys Peak in Colorado in honor of Devin Argall and the muscular dystrophy community...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Getzlaf Golf Shootout, September 12-13, 2015, Costa Mesa and Dana Point, Calif.

The 5th Annual Getzlaf Golf Shootout hosted by Ryan Getzlaf, captain of the Anaheim Ducks is a two-day charity golf event that brings together athletes, celebrities and community leaders, all teaming up in support of CureDuchenne...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Continuing Education Course for Physical Therapists, September 18, Chattanooga, TN

This course is ideal for physical therapists, occupational therapists, adapted physical educators and would be beneficial for others involved in the care of persons with Duchenne such as speech and language pathologists, educators, physicians, and nursing professionals...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Duchenne Families, September 19, Chattanooga, TN

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on September 19 in Chattanooga, TN. The session provides updates on stretching, equipment, activity and school modification, mobility management, research and clinical trials...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

All in for Duchenne, October 3, 2015, Sylvania, OH

The 3rd Annual All in for Duchenne to benefit Braedan’s Bridge. 100% of the proceeds from this event will fund research to find a cure for Duchenne muscular dystrophy. This sports-themed event held in a sports complex field house will feature games, raffles, silent auction, dinner by Jed’s Barbeque and Brew and much more!...

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