August 2015
Together we can Cure Duchenne!
Research
Duchenne Research Highlights from MDA Conference

Sarepta Therapeutics Announces FDA Has Filed Eteplirsen NDA for the Potential Treatment of Duchenne Muscular Dystrophy for Patients Amenable to Exon 51 Skipping

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, announced August 25 that the U.S. Food and Drug Administration (FDA) has filed the New Drug Application (NDA) for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Approximately 13% of people with Duchenne muscular dystrophy are estimated to have a mutation addressable by Eteplirsen/exon 51 skipping....

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Duchenne Research Highlights from MDA Conference

Duchenne Community: Let Your Voice Be Heard!

This week the FDA accepted Sarepta Therapeutics’ New Drug Application for eteplirsen with priority review. This follows on the June FDA’s acceptance of BioMarin’s NDA for drisapersen, also with priority review...

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Duchenne Research Highlights from MDA Conference

Pfizer Currently Recruiting Participants for Phase 2 Clinical trial on Duchenne Muscular Dystrophy

Pfizer is currently recruiting participants to enroll a phase 2 clinical trial that intends to assess the safety, efficacy and pharmacological parameters of its product, PF-06252616, in boys with Duchenne muscular dystrophy (DMD) who are able to walk (ambulatory)...

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Duchenne Research Highlights from MDA Conference

BioMarin Quarterly Update to the Duchenne Community

In an effort to provide regular communication on our DMD programs to the Duchenne community, we are circulating a quarterly update. This update is the first of its kind for BioMarin, and we welcome your feedback to this new communication channel, which you may do by contacting BioMarin Patient Advocacy...

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Duchenne Research Highlights from MDA Conference

BioMarin and Sarepta Receive Rare Pediatric Disease Designation from FDA

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for drisapersen and eteplirsen, potential treatments for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to exon 51 skipping treatment...

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

Fibrogen Receives FDA Clearance to Proceed with Clinical Study of FG-3019 in Duchenne Muscular Dystrophy

FibroGen, Inc. (Nasdaq:FGEN), a research-based biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has completed its review of the Company's investigational new drug (IND) application for the study of FG-3019 in patients with Duchenne muscular dystrophy (DMD), and clinical study may proceed...

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CureDuchenne Cares
Abilities Expo Video Blog

Duchenne Workshops

CureDuchenne Cares had a successful workshop in Minneapolis on August 14 and 15. Physical therapists, parents and caregivers were updated on the best practices for physical therapy, Duchenne care, research and clinical trials...

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Abilities Expo Video Blog

August Video Blog

Those with Duchenne muscular dystrophy require modifications in their school environment, especially when it comes to PE class...

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

11-Year-Old Tyler Armstrong Plans to Be the Youngest to Climb Mount Everest to Help Find a Cure for Duchenne Muscular Dystrophy

Tyler Armstrong began climbing mountains at seven years old for a fun challenge. Now at 11, Tyler climbs for those who can't, the 300,000 boys around the world with Duchenne muscular dystrophy in the hopes of raising $1 million dollars to help find a cure...

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Upcoming Events
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...

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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

CureDuchenne Cares Continuing Education Course for Physical Therapists, September 18, Chattanooga, TN

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, September 19, Chattanooga, TN

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on September 19 in Chattanooga, TN...

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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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