June 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

Capricor Announces Receipt of FDA Clearance for the Phase I/II Study of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy Related Cardiomyopathy

Capricor Therapeutics, Inc. (Nasdaq:CAPR), a biotechnology company focused on developing novel therapeutics for the treatment of cardiovascular diseases, announced on June 8 that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug Application (IND) for the clinical development of CAP-1002...

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

Capricor Webinar on HOPE-DUCHENNE Trial

Capricor Therapeutics and CureDuchenne hosted a webinar on June 19. The presentation discussed the HOPE-DUCHENNE Trial...

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Duchenne Research Highlights from MDA Conference

BioMarin Submits Drisapersen MAA to EMA for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced June 8 the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for drisapersen, an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping...

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Duchenne Research Highlights from MDA Conference

Akashi Therapeutics Reports Positive Clinical Data on HT-100 in Patients with Duchenne Muscular Dystrophy

Akashi Therapeutics Inc., a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD), today announced positive interim clinical data from an ongoing Phase 1b/2a clinical program with HT-100 (delayed-release halofuginone) an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in boys with DMD...

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Duchenne Research Highlights from MDA Conference

Santhera Update on Pre-NDA Meeting with FDA on RaxoneŽ/CatenaŽ in Duchenne Muscular Dystrophy (DMD)

Santhera Pharmaceuticals (SIX: SANN) announces on June 10 that it has discussed its plans to submit a New Drug Application (NDA) for RaxoneŽ/CatenaŽ (idebenone) in DMD with the US FDA in a pre-NDA meeting...

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Duchenne Research Highlights from MDA Conference

Catabasis Pharmaceuticals Initiates MoveDMD a Phase 1/2 Trial of CAT-1004 for the Treatment of Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals Inc. a clinical-stage drug development company built on a pathway pharmacology technology platform announced on June 17 that dosing of the first patient has been initiated in the MoveDMD trial a Phase 1/2 trial of CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD)...

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Duchenne Research Highlights from MDA Conference

Gene controls stem cells during muscle regeneration

Unlike many other organs, skeletal muscles have a high potential for regeneration. When a muscle is injured, the muscle stem cells also known as satellite cells located between the individual muscle fibres rapidly begin to proliferate and subsequently replace the damaged muscles cells...

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Duchenne Research Highlights from MDA Conference

CDMRP Announced Funding Opportunities for DMD

The Congressionally Directed Medical Research Programs has announced funding opportunities for the Duchenne Muscular Dystrophy Research Program. CureDuchenne founder Debra Miller has been on the review committee since its inception...

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CureDuchenne Cares
Abilities Expo Video Blog

PT’s, Parents and Caregivers Benefit from CureDuchenne Cares Workshops in San Diego

Physical therapists, parents and caregivers gained valuable information about Duchenne care and the latest Duchenne research at the CureDuchenne Cares workshops in San Diego on June 5 and 6. The session was hosted by the San Diego Muscle Research Center and held at the Sanford-Burnham Medical Research...

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Abilities Expo Video Blog

New Video Blog: Exercise Study in Duchenne

The latest CureDuchenne Cares video discusses the No Use Is Disuse study published in 2013 by M. Jansen which describes the bicycle exercise routine of boys with Duchenne and the benefits found over a six month period...

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

Military, Letter Carriers and other Federal Employees Give Hope through Workplace Campaign

CureDuchenne is proud to announce its designation as a Combined Federal Campaign (CFC) approved charitable organization. The CFC provides a simple way for federal employees to donate to CureDuchenne through automatic payroll donations...

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

Revell Family Featured in Austin American-Statesman

Bursting with energy, Timothy and Andrew Revell are all over their Leander house. Their schoolwork is spread out on a counter near the kitchen, where their mother, Laura Revell, has been leading the boys through their lessons...

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

Hero Dads - Ryan Getzlaf Featured in Parenting OC Magazine

Ryan Getzlaf doesn’t have much free time as the captain of the Anaheim Ducks. He’s also busy parenting two young boys and a baby daughter with his wife, Paige, and raising money for a charity that funds research to find a cure for Duchenne muscular dystrophy...

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Upcoming Events
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

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. Devin is a 13 year old boy who has Duchenne muscular dystrophy...

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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!...

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