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

Sarepta Therapeutics Announces Additional Long-Term Efficacy and Safety Data from Pivotal Phase IIb Program of Eteplirsen for Treatment of Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, announced on October 1additional clinical efficacy and safety data from the Company’s Phase IIb program of eteplirsen in patients with Duchenne muscular dystrophy (DMD)...

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Duchenne Research Highlights from MDA Conference

BioMarin Announces FDA Advisory Committee to Review Drisapersen for Treatment of Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review the New Drug Application (NDA) for drisapersen...

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Duchenne Research Highlights from MDA Conference

Sarepta Therapeutics Announces Tentative FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) is tentatively scheduled to review Sarepta’s New Drug Application (NDA) for eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, on January 22, 2016...

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Speak Up for the Duchenne Community!

After waiting for a very long time, the Duchenne community now has clarity on the Advisory Committee meetings dates for BioMarin and Sarepta. BioMarin’s date is November 24, 2015 and Sarepta’s date is January 22, 2016...

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PTC Announces Results from Phase 3 ACT DMD Clinical Trial of Translarna™ (ataluren) in Patients with Duchenne Muscular Dystrophy

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced results from the Phase 3, double-blind, placebo-controlled, 48-week ACT DMD trial of Translarna™ (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD)...

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European Commission Grants Orphan Medicinal Product Designation for CAT-1004, Catabasis Pharmaceuticals’ Investigational Therapy 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 October 26 that the European Commission (EC) has granted orphan medicinal product designation to CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD)...

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Gene Therapy Could Treat Debilitating Form Of Muscular Dystrophy

Duchenne muscular dystrophy is a debilitating genetic disorder that has garnered much attention lately as the U.S. Food and Drug Administration prepares to review two experimental drugs that could soon treat some patients with the degenerative muscle disease...

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Muscular Dystrophy: Manipulating Cell Signaling for Better Muscle Function

A team of researchers at the University of Michigan Health System identified a novel method of triggering the "instructions" normally given to cells by the muscle protein dystrophin, which is found in cardiac muscles cells and in muscles used for movement...

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Capricor Therapeutics and CureDuchenne to Host Webinar on November 3, 2015 at 3:00 p.m. ET

Capricor Therapeutics, Inc. (Nasdaq: CAPR), a biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics and CureDuchenne, a national nonprofit organization dedicated to finding a cure for Duchenne muscular dystrophy, invite participants to join a webinar “HOPE-Duchenne: A Clinical Trial for Individuals with Heart Disease Related to DMD” on Tuesday, November 3, 2015 at 3:00 p.m. ET/noon PT...

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CureDuchenne Cares
Abilities Expo Video Blog

VIDEO BLOG: Transferring Someone with Duchenne

For someone with Duchenne, moving from one position to another can become very difficult or impossible without the assistance of someone else or specialized equipment...

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Abilities Expo Video Blog

Redefining the Standard of Care for Duchenne Muscular Dystrophy: Child Neurology Symposium

PeerView Institute for Medical Education and CureDuchenne hosted a medical education symposium on Duchenne muscular dystrophy on October 8. The symposium “Redefining the Standard of Care for Duchenne Muscular Dystrophy: What is the potential role of emerging therapies?” was held during the Child Neurology Society annual meeting in National Harbor, Maryland...

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Abilities Expo Video Blog

A lot of Robust Discussion, Interaction at CureDuchenne Cares' October Workshop

With the recent announcement of the Advisory Committee meeting for BioMarin and Sarepta Therapeutics, there has been a lot of excitement and speculation of what is to come for the Duchenne community...

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

Devastating Disease Bridges Borders Between Duchenne Community in California and Nepal

Whether you live in the paradise of Southern California or the cold mountains of Nepal, the physical and emotional challenges of Duchenne muscular dystrophy are the same. This lethal genetic disease causes muscle deterioration that reduces mobility and motor function as patients get older, and claims their lives by their mid-20s...

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

Ain't no mountain high enough ... so says this 11-year-old

Eleven-year-old Tyler Armstrong of Yorba Linda is gearing up to become the youngest person to climb Mount Everest the world’s tallest mountain...

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Recent Success
CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

All in for Duchenne

The 3rd Annual All in for Duchenne event to benefit Braedan’s Bridge was held Saturday, October 3, 2015, at Sylvania Tam-O-Shanter in Sylvania, OH. Braedan’s Bridge is a family founded non-profit organization established in honor of Braedan Henegar, who lives with Duchenne...

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Upcoming Events
CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

HOPE-Duchenne: A Clinical Trial for Individuals with Heart Disease Related to DMD Webinar, November 3

Capricor Therapeutics, Inc. and CureDuchenne are hosting a webinar “HOPE-Duchenne: A Clinical Trial for Individuals with Heart Disease Related to DMD” on Tuesday, November 3, 2015 at 3:00 p.m. ET/noon PT...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Napa in Newport, February 27, 2016, St. Regis Hotel, Dana Point, CA

Napa in Newport is Southern California's premier wine event. Napa in Newport brings together as many as 40 of Napa's best wineries and world class chef for a truly unforgettable experience...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Champions to CureDuchenne Austin, April 9, 2016, Austin, TX

Save the date for the Champions to CureDuchenne Austin event on April 9, 2016 in Austin, TX. This annual event will benefit CureDuchenne and is presented by Johnny Carino’s...

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