July 2016
Together we can Cure Duchenne!
Research
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An Update for the Duchenne Community

Sarepta Therapeutics is updating the Duchenne community regarding recent changes to the Phase III confirmatory clinical study, ESSENCE (Study 4045-301), as posted on clinicaltrials.gov. [Identifier: NCT02500381]...

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BioMarin Update for the Duchenne Community: 7th July, 2016

BioMarin is providing a further update to the Duchenne Community following our announcement of the withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on compounds BMN 044, BMN 045 and BMN 053...

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Capricor Therapeutics Provides Enrollment Update on HOPE Clinical Trial in Duchenne Muscular Dystrophy

Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics, announced on June 23 that patient enrollment in its ongoing randomized HOPE-Duchenne clinical trial (Halt cardiomyOPathy progrEssion in Duchenne) has exceeded 50% of its 24-patient target...

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PTC Initiates Phase 2 Clinical Trial of Translarna (ataluren) for Treatment of Nonsense Mutation Duchenne Muscular Dystrophy in Pediatric Patients Between the Ages of Two and Five Years

We are pleased to inform you that PTC Therapeutics has initiated a Phase 2 clinical trial of Translarna™ (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD), in patients between the ages of two and five (they cannot have turned five to participate)...

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Catabasis Pharmaceuticals Announces the Initiation of an Open-Label Extension for the MoveDMD Trial Studying Edasalonexent (CAT-1004) in Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced on July 7 the initiation of an open-label extension for the Phase 2 portion (Part B) of the MoveDMD trial studying edasalonexent (CAT-1004), an investigational therapy, in boys with Duchenne muscular dystrophy (DMD)...

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NHS England Enables Access to Translarna™ ▼ (ataluren) For Patients with Nonsense Mutation Duchenne Muscular Dystrophy

Important decision allows reimbursed access to Translarna, the first approved therapy to treat the underlying cause of Duchenne muscular dystrophy...

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Santhera Updates on U.S. Regulatory Filing for RaxoneŽ (idebenone) in Duchenne Muscular Dystrophy (DMD)

Santhera Pharmaceuticals (SIX: SANN) announces that it has received written correspondence from the U.S. Food and Drug Administration (FDA) on its proposed subpart H approval pathway for Raxone in DMD patients not taking concomitant glucocorticoids...

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Catabasis Quarterly Update on MoveDMD

We are pleased to share the 4th issue of the MoveDMD trial newsletter from Catabasis, highlighting the open-label extension for the MoveDMD trial and a summary of the findings to date as well a link to the recent webinar...

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Pluristem Reports Data Showing PLX-PAD Cells Effective in Treating Duchenne Muscular Dystrophy

Pluristem Therapeutics Inc. (Nasdaq:PSTI), (TASE:PSTI)/(PLTR), a leading developer of placenta-based cell therapy products, reported on June 22 positive data from preclinical studies of its PLX-PAD cells in the treatment of Duchenne muscular dystrophy...

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CureDuchenne Cares
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Windy City Welcomes CureDuchenne Cares

The first of two July CureDuchenne Cares sessions took place on July 9th at Vital Rehabilitation, a multidisciplinary outreach therapy clinic in a charming part of Chicago. It was a perfect location that drew in families from not only Illinois, but also Indiana, Wisconsin, and Michigan...

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

Knoxville Brewfest Benefits CureDuchenne

On June 18, 2016, 2,500 beer enthusiasts gathered near the Southern Railway Terminal in downtown Knoxville, Tennessee, to participate in the 6<sup>th</sup> annual Knoxville Brewfest...

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

12-Year-Old Tyler Armstrong Conquers Mt. Denali

On June 23, 12-year-old Tyler Armstrong reached the summit of Mt. Denali, becoming the sixth youngest person to do, and coming one step closer to his goal of completing the Seven Summits in his Climb to CureDuchenne effort...

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Upcoming Events
CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, July 30, Sacramento, CA

Please join CureDuchenne for a free, informative class on muscular dystrophy on July 30 in Sacramento, CA for family members and caregivers at Shriner’s Hospital for Children, 2425 Stockton Blvd, Sacramento from 8:30 a.m. 2 p.m...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Getzlaf Golf Shootout, August 26 and 27, Costa Mesa and Dana Point, CA

The 6th 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 Workshop for Families, September 24, Charlotte, NC

Please join CureDuchenne for a free, informative class on muscular dystrophy on September 24 in Charlotte, NC for family members and caregivers at Sheraton Charlotte Airport Hotel, 3315 Scott Futrell Drive, Charlotte, NC, 28208 from 8:30 a.m. to 2 p.m...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

All in for Duchenne, October 15, Sylvania, OH

The annual All in for Duchenne event benefiting CureDuchenne is on October 15 from 6 p.m. to 11 pm. Tickets include admission to the event, dinner by Jeds Barbeque and Brew, drinks and chance to win in the reverse raffle. Proceeds of the event will help fund Duchenne muscular dystrophy research...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Dealing for Duchenne, October 15, San Antonio, TX

Join us for a night of hope at the Dealing for Duchenne San Antonio on October 15...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Dealing for Duchenne, October 20, Cambridge, MA

A Vegas gaming night to benefit the cure for Duchenne will be held on October 20 from 6 p.m. to 10 p.m. at 650 East Kendall Street in Cambridge, MA...

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