May 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

Sarepta Therapeutics Announces Plans to Submit Rolling NDA for Eteplirsen following Today’s Pre-NDA Meeting with the FDA

Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-targeted therapeutics, announced on May 19 that the Company held a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) regarding its lead product candidate, eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD)...

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

Capricor Therapeutics has filed an IND with the FDA

Capricor Therapeutics has filed an IND with the FDA for a clinical trial for Duchenne with its cardiosphere-derived cells (CDC). In the mdx mouse model of Duchenne, these cells showed improvement in cardiac function and exercise performance...

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Duchenne Research Highlights from MDA Conference

Clinical phenotypes as predictors of the outcome of skipping around DMDexon 45

When CureDuchenne first funded Prosensa 11 years ago, the original plan was to develop an exon 46 skipping drug for those patients with a deletion of exon 45. Fortunately, the scientists at Prosensa switched to skipping exon 44 for the reasons in this very informative paper...

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CureDuchenne Cares
CureDuchenne Cares Debuts in Toledo, Ohio

Pharmaceutical Companies Support CureDuchenne Cares to Provide Standard of Care for Duchenne Muscular Dystrophy

Three pharmaceutical companies have partnered with CureDuchenne to support CureDuchenne Cares, a physical therapy, education and outreach program for the Duchenne muscular dystrophy community to help establish and provide consistent standard of care for Duchenne patients...

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Abilities Expo Video Blog

Passion for Physical Therapy and Duchenne Care

Guest blog by Jennifer Wallace, physical therapist and project manager of CureDuchenne Cares. Yesterday was an important day for me since it was my last day working in a local pediatric physical therapy clinic...

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Abilities Expo Video Blog

New Video Blog: Wrist, Hand Orthosis (Brace)

Those with Duchenne muscular dystrophy can experience significant muscle stiffness and joint contractures in the wrist, hand and fingers. Wrist-Hand-Orthoses can be used to improve resting position, increase range of motion / flexibility and improve hand use...

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News
CureDuchenne Honors Dr. Brenda Wong from Cincinnati Children’s Hospital with CureDuchenne Cares Award

Treatments on the Horizon

The Duchenne community is celebrating another promising step toward an approved treatment. On May 19 Sarepta Therapeutics announced plans to submit a rolling NDA for eteplirsen with the final NDA submission landing in the FDA’s hands by mid-year...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

BioWorld

CureDuchenne is prominently featured in a BioWorld Today article on BioMarin’s rolling new drug application (NDA). It highlights several research projects CureDuchenne has funded...

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Biotech’s Path to Discovery: BioMarin CEO

OC Family

Thank you to inspiring moms like Paige Getzlaf for supporting CureDuchenne! Read the article in OC Family on page 55. This year's Getzlaf Golf Shootout is Sept. 12-13...

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Celebrity Hosts Join Second Annual Blingo to CureDuchenne

Riviera

The Napa in Newport wine auction to benefit CureDuchenne is featured in the May issue of Riviera Magazine. Cheers...

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

Anaheim Ducks Captain Ryan Getzlaf to Host 5th Annual Getzlaf Golf Shootout to Benefit Cure Duchenne

The 5th Annual Getzlaf Golf Shootout benefiting CureDuchenne will be held September 12 at Sutra Lounge in Costa Mesa, Calif. and September 13 at the Monarch Beach Golf Links in Dana Point, Calif...

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

Mack Brown, Jack Ingram, and Matthew McConaughey Announce Over 1.5 Million Dollars Raised by MJ&M 2015. 2016 Event to be held April 14-15th

Mack, Jack & McConaughey (MJ&M), the joint fundraising effort of ESPN analyst and legendary football coach Mack Brown, ACM award winning recording artist Jack Ingram, and Academy AwardŽ-winning actor Matthew McConaughey, announced on May 11 that their third annual event raised over $1.5 million last month to benefit a handful of organizations that all reflect MJ&M’s goal to empower kids...

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

Champions Fighting to CureDuchenne

For Some People It Takes A Trophy Or Medal To Denote A Champion. For Those In The Fight Against Duchenne Muscular Dystrophy, There Are All Kinds Of Champions And You Won’t Know Them By Their Trophy...

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Upcoming Events
CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Continuing Education Course for Physical Therapists, June 5, 2015, San Diego

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, June 6, 2015, San Diego

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on June 6 in San Diego, Calif. 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

Knoxville Brewfest, June 20, 2015, Knoxville, Tenn.

The 5th Annual Knoxville Brewfest will be held on June 20 to benefit CureDuchenne. It will be held from 4 p.m. - 8 p.m. near the Southern Railway Station on the Gay Street viaduct and Depot Avenue in downtown Knoxville...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Paddle Battle, June 27, 2015, Los Angeles

Join us for CureDuchenne’s Paddle Battle, a celebrity table tennis tournament to help fund research to find a cure for Duchenne muscular dystrophy...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Continuing Education Course for Physical Therapists, June 26, 2015 Long Beach, Calif.

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, June 27, 2015 Pasadena, Calif.

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on June 27 in Pasadena, Calif. 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

CureDuchenne Cares Continuing Education Course for Physical Therapists, July 17, 2015, Maple Valley, Washington

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, July 18, 2015, Maple Valley, Washington

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on July 18 in Maple Valley, Washington. 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

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

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! A few new attractions are being added for this year as well...

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