April 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 Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced on April 27 the completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for drisapersen, an investigational exon-skipping drug candidate for the treatment of the largest genetically defined subset of Duchenne muscular dystrophy (DMD)...

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Duchenne Research Highlights from MDA Conference

Sarepta Therapeutics Appoints Edward Kaye, M.D., as Interim CEO; Company on Track with Clinical and Regulatory Plans for Investigational Duchenne Muscular Dystrophy Drugs

Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-targeted therapeutics, today announced the appointment of Edward Kaye, M.D., the company’s Chief Medical Officer (CMO), as interim Chief Executive Officer (CEO) effective immediately...

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Duchenne Research Highlights from MDA Conference

Capricor Granted FDA Orphan Drug Designation for Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy

CureDuchenne Ventures provided significant funding for Capricor Therapeutics to advance their CDC cells for cardiac treatment in Duchenne. CureDuchenne continues to be the innovator in Duchenne research by aggressively tackling the primary cause of death in Duchenne patients...

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Duchenne Research Highlights from MDA Conference

Santhera's Positive Phase III Trial (DELOS) in Patients with Duchenne Muscular Dystrophy Published in The Lancet

Santhera Pharmaceuticals (SIX: SANN) announced that the full results of the double-blind placebo-controlled Phase III trial (DELOS) demonstrating efficacy and safety of Raxone®/Catena® (INN: idebenone) in patients with DMD have been published in The Lancet (Lancet 2015; 385: 1748-57. Online publication: http://dx.doi.org/10.1016/S0140-6736(15)60025-3)...

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Duchenne Research Highlights from MDA Conference

PhaseBio Announces Promising Pre-Clinical Results for PB1046 in Models of Duchenne Muscular Dystrophy

PhaseBio Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company and leader in the field of biopolymer-based drugs focused on developing treatments for metabolic and specialty cardiopulmonary disorders, announced that positive pre-clinical data on its compound PB1046 in Duchenne muscular dystrophy (DMD) models will be presented today in the emerging science poster session at the 67th American Academy of Neurology (AAN) Annual Meeting being held at the Walter E. Washington Convention Center in Washington, DC...

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Duchenne Research Highlights from MDA Conference

Myocardial Fibrosis Burden Predicts Left Ventricular Ejection Fraction and Is Associated With Age and Steroid Treatment Duration in Duchenne Muscular Dystrophy

Patients with Duchenne muscular dystrophy exhibit progressive cardiac and skeletal muscle dysfunction. Based on prior data, cardiac dysfunction in Duchenne muscular dystrophy patients may be influenced by myocardial fibrosis and steroid therapy...

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Duchenne Research Highlights from MDA Conference

Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.

Exon-skipping therapies aim to convert Duchenne muscular dystrophy (DMD) into less severe Becker muscular dystrophy (BMD) by altering pre-mRNA splicing to restore an open reading frame, allowing translation of an internally deleted and partially functional dystrophin protein...

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Duchenne Research Highlights from MDA Conference

VBP15 To Treat Duchenne Muscular Dystrophy - Clinical Trial Update

In this interview, Eric Hoffman, PhD, president and chief executive officer of ReveraGen Biopharma, provides an update on the phase 1 and 2 studies either underway or planned for the use of VPM15 to treat patients with Duchenne muscular dystrophy...

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Duchenne Research Highlights from MDA Conference

In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy

The use of antisense ‘splice-switching’ oligonucleotides to induce exon skipping represents a potential therapeutic approach to various human genetic diseases...

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CureDuchenne Cares
CureDuchenne Cares Debuts in Toledo, Ohio

CureDuchenne Cares

CureDuchenne Cares presents the latest video blog in the Duchenne education series. The Alter G antigravity treadmill is a good way for those with Duchenne muscular dystrophy to walk in a safe environment...

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Abilities Expo Video Blog

The PJ Nicholoff Steroid Protocol

CureDuchenne Cares is pleased to share important information about steroids use for Duchenne patients...

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

Hope and Gratitude for Moving Closer to a Cure for Duchenne

As I type this, my heart is filled with hope. A horizon that we could barely see 12 years ago has arrived. When our son Hawken was diagnosed with Duchenne Muscular Dystrophy 12 years ago, we felt lost and scared...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

The Power of Parents

CureDuchenne was featured in the May issue of Parents Magazine. It features Debra and Paul Miller and their journey to find a cure for Duchenne muscular dystrophy...

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Biotech’s Path to Discovery: BioMarin CEO

Carve to CureDuchenne

On Saturday, May 2, 2015, Bill and Kim Procko are hosting “Carve to CureDuchenne,” a skateboarding contest to benefit CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy...

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Celebrity Hosts Join Second Annual Blingo to CureDuchenne

Make Mom Smile

Mother's Day is Sunday, May 10. Buy your Mother’s Day Gifts at smile.Amazon.com and Amazon will donate to CureDuchenne...

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

Vince Young Helps CureDuchenne Raise More than $300,000 to Find a Cure for Duchenne Muscular Dystrophy

In 2005 while Vince Young was leading his University of Texas teammates to a coveted national title, the Revell family in Austin was learning that their young son had Duchenne Muscular Dystrophy and might be wheelchair-bound by the time he was in college...

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

Blingo Raised More Than $180,000 for Duchenne Research

“Blingo!” was the sound of the evening during the second annual Blingo to CureDuchenne fundraiser on Saturday, April 11 at the Rittenhouse Hotel...

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MJ&M Empowers Kids

MJ&M Empowers Kids

Once again Mack Brown, Jack Ingram and Matthew McConaughey lit up downtown Austin for the MJ&M charity event in Austin on April 16 and 17...

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Upcoming Events
Support CureDuchenne in the iheartoc Giving Day

Carve to CureDuchenne, May 2, 2015 Branford, Florida

On Saturday, May 2 skateboarders from across Florida will take part in this one of a kind, heartfelt event, raising money for research to cure a 100% fatal form of muscular dystrophy called, “Duchenne”. The skateboarder who completes the most consecutive wall to wall turns, or , “carves”, in the skateboard course known as a bowl, (similar to an empty swimming pool), will earn your pledge dollars for muscular dystrophy research...

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Tickets on Sale for the 5th Annual Knoxville Brewfest

Laugh For A Cure, May 7, 2015 Royal Oak, MI

Laugh for a Cure will be held on Saturday, May 7 at Mark Ridley’s comedy Castle in Royal Oak, MI at 7pm. Comedian Josh Wolf from Comedy Central performing...

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Napa In Newport Raises $680,000 To Help Save The Lives of Boys With Duchenne Muscular Dystrophy

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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Napa In Newport Raises $680,000 To Help Save The Lives of Boys With Duchenne Muscular Dystrophy

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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Champions to CureDuchenne, April 11, 2015, Austin, Texas

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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Blingo to CureDuchenne, April 11, 2015, Philadelphia

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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MJ&M, April 16-17, 2015 Austin, Texas

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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