August 2016
Together we can Cure Duchenne!
Research
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Bamboo Therapeutics Acquired by Pfizer Inc., Early Stage Investment by CureDuchenne Ventures, LLC Enabled Bamboo Therapeutics to Accelerate Development of a Gene Therapy for Duchenne Muscular Dystrophy

On August 1, Pfizer Inc. announced its acquisition of Bamboo Therapeutics in a deal worth up to $645 million. Bamboo Therapeutics is an early-stage biotechnology company developing therapies for rare neuromuscular and central nervous system diseases...

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PTC Therapeutics Provides Regulatory Update on Translarna™ (ataluren)

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced on July 25 global regulatory updates on Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) and nonsense mutation cystic fibrosis (nmCF)...

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Sarepta Recruiting for ESSENCE Trial

Sarepta Therapeutics is now recruiting for their exon 45 and exon 53 studies: For trial sites and inclusion criteria...

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PhaseBio Announces Dosing of First Patients in Two-Part Phase 2a Study of PB1046 in Cardiopulmonary Disorders

PhaseBio Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on biopolymer-based drugs for orphan, cardiopulmonary and metabolic diseases, announced on August 8 the dosing of the first patients in Part One of a Phase 2a clinical study of PB1046, a once-weekly vasoactive intestinal peptide (VIP) receptor agonist in development for the treatment of cardiopulmonary disorders...

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FDA Accepts Marathon Pharmaceuticals’ New Drug Applications for Deflazacort for the Treatment of Duchenne Muscular Dystrophy and Grants Priority Review

Marathon Pharmaceuticals, LLC, a biopharmaceutical company developing treatments for rare diseases, announced August 10 the New Drug Applications (NDA) for the investigational drug deflazacort have been accepted for filing and granted Priority Review by the U.S. Food and Drug Administration (FDA)...

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Tivorsan Pharmaceuticals Granted FDA Orphan Drug Designation for Human Recombinant Biglycan in the Treatment of Duchenne Muscular Dystrophy

Tivorsan Pharmaceuticals, Inc., a biotechnology company that is pioneering a novel approach to treating all forms of Duchenne Muscular Dystrophy (DMD) and other serious, debilitating neuromuscular disorders, announced on August 15 that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead investigational drug, human recombinant Biglycan, for the treatment of DMD...

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CureDuchenne Cares
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CureDuchenne Cares Joins Forces with UC Davis for Special Session

On Saturday, July 30, CureDuchenne Cares completed its ninth family and caregiver workshop of the year, bringing a unique educational experience to 20 Duchenne family members and patients in Sacramento, CA...

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CureDuchenne Announces Unique, Educational Event for Families

Currently with no cure, education on various care and management practices are very important to extend quality of life for developing children with Duchenne muscular dystrophy. Stepping outside of the successful CureDuchenne Cares Family and Caregiver workshop mold, this unique educational event will bring additional topics to Duchenne families on the west coast, including...

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

Anaheim Ducks Captain Ryan Getzlaf to Host 6th Annual Getzlaf Golf Shootout to Benefit CureDuchenne

The 6th Annual Getzlaf Golf Shootout presented by ActivePDF, benefiting CureDuchenne will be held Friday, August 26 at Sutra Lounge in Costa Mesa, Calif., and Saturday, August 27 at the Monarch Beach Golf Links in Dana Point, Calif...

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

New Voices in Medical Advocacy Often Are Patients

Parent-advocates of children with chronic conditions have long worked toward finding cures; adult self-advocates are shifting the focus to goals of independent work and living...

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

Pfizer plucks Bamboo rAAV technology in potential $645M gene therapy play

CureDuchenne Founder Debra Miller is quoted in this BioWorld article about Pfizer acquiring Bamboo...

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

Climbing for Those Can’t

Tyler Armstrong was featured on the Today Show on an “Extreme Kids” segment.  Tyler has been climbing for all those with Duchenne muscular dystrophy.  We are grateful for Tyler’s compassion for those that are less physically enabled...

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Upcoming Events
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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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Family Summit, October 22, Garden Grove, CA

Join us for the CureDuchenne Cares Family Summit on October 22.  It is a free educational event for families who have loved ones with Duchenne muscular dystrophy...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Napa In Newport, March 4, 2017, Laguna, Nigel, CA

Reserve the date for Napa In Newport on March 4, 2017 at the Ritz-Carlton, Laguna Niguel.  Napa In Newport brings together 40 of Napa’s most distinguished wineries to Orange County for an extraordinary wine and culinary experience...

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