April 2016
Together we can Cure Duchenne!
Research
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Sarepta Issues Statement on Advisory Committee Outcome for Use of Eteplirsen in the Treatment of Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-based therapeutics, announced on April 25 that the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Advisory Committee (PCNSC) met to review the new drug application (NDA) for eteplirsen as a treatment for Duchenne muscular dystrophy amenable to exon 51 skipping...

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CureDuchenne Responds to FDA Advisory Committee's Recommendation Against Approval of Eteplirsen for Duchenne Muscular Dystrophy

CureDuchenne, the California-based nonprofit organization dedicated to finding a cure for every person with Duchenne muscular dystrophy, has released the following statement from its co-founder and CEO, Debra Miller, following the recommendation against approval by the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) for Sarepta’s exon-skipping drug eteplirsen...

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Capricor Therapeutics Announces DSMB Recommends Continuation of HOPE-Duchenne Clinical Trial

Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics, announced on April 4 that the independent Data Safety Monitoring Board (DSMB) of the Phase I/II HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) clinical trial has completed its pre-specified review of the first patient cohort...

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Summit Reports Positive Interim Data from Ongoing Phase 1 Clinical Trial Testing a New Formulation of SMT C1100 in Patients with DMD

Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') and Clostridium difficile infection, announces positive interim data from a Phase 1 clinical trial evaluating a new oral formulation of its lead utrophin modulator, SMT C1100, for DMD...

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Duchenne Research Highlights from MDA Conference

New Research Partnership Aims to Accelerate New Drug for Fatal Childhood Muscle Disease

A prominent disease research foundation has invested in RASRx, a startup company formed by USC researchers working to develop a novel therapy for Duchenne, a rare and deadly form of muscular dystrophy...

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Duchenne Research Highlights from MDA Conference

Catabasis Pharmaceuticals Initiates Part B of the MoveDMD Trial of CAT-1004 for the Treatment of Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced that dosing of the first patient has been initiated in Part B of the MoveDMD trial, a 12-week trial to assess the efficacy of CAT-1004 in Duchenne muscular dystrophy (DMD)...

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Duchenne Research Highlights from MDA Conference

NICE Recommends Translarna™ (ataluren) for the Treatment of Patients with Nonsense Mutation Duchenne Muscular Dystrophy in England

PTC Therapeutics, Inc. (NASDAQ: PTCT), announced that the National Institute for Health and Care Excellence (NICE) has recommended Translarna ∇ (ataluren) for ambulatory patients aged five years and older with nonsense mutation Duchenne muscular dystrophy (nmDMD) in connection with a Managed Access Agreement (MAA) with NHS England...

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Duchenne Research Highlights from MDA Conference

Marathon Pharmaceuticals Presents Analyses at AAN of a Phase III Clinical Study of Deflazacort in Patients with Duchenne Muscular Dystrophy (DMD)

Marathon Pharmaceuticals, a biopharmaceutical company focused solely on finding treatments for rare diseases, announced presentations of analyses of a pivotal Phase III study of deflazacort, an investigational glucocorticoid, in patients with Duchenne Muscular Dystrophy (DMD)...

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CureDuchenne Cares
Abilities Expo Video Blog

New Nutrition Video

Nutrition and proper diet are very important for those who have Duchenne. Check out the newest CureDuchenne Cares video on nutrition considerations for those with Duchenne...

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Abilities Expo Video Blog

Cares Sessions Provide Education, Demonstration and Discussion

What a busy month April has been for CureDuchenne Cares and the Duchenne community as a whole! Our organization wrapped up two successful CureDuchenne Cares workshops: one in Houston, TX and the other in Kansas City, MO, announced one of our next locations this year (Long Island, NY in June 2016), and welcomed an important addition to the team, Doug Levine, PT...

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

Media Coverage on Advisory Committee

There was a lot of media coverage surrounding the FDA Advisory Committee meeting for Sarepta. Here is a selection of news articles pre-, during and post the Advisory Committee meeting that featured a quote and perspective from Debra Miller, founder and CEO of CureDuchenne...

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

Blingo to CureDuchenne Returns for its 3rd Annual Fundraiser

CureDuchenne, a national nonprofit that raises awareness and funds research to find a cure for Duchenne Muscular Dystrophy, will put the bling in bingo for their third annual fundraiser on Saturday, April 30th, 2016 at The Logan Hotel...

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

12-year-old Record-Breaking Mountaineer Tyler Armstrong Sets Sights on Summit #4 - Australia’s Mt. Kosciuszko To Raise Funds for CureDuchenne

In his quest to climb the Seven Summits to help raise awareness and help CureDuchenne find a cure for Duchenne muscular dystrophy, record-breaking mountaineer Tyler Armstrong has now set his sights on Mt. Kosciuszko, Australia’s highest peak, as his fourth summit...

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

Texans Take Aim on a Cure for Duchenne Muscular Dystrophy

On April 9, hundreds of big-hearted Texans, along with former University of Texas football player Vince Young and KXAN weathercaster Jim Spencer, gathered at The University of Texas Golf Club for the seventh annual Champions to CureDuchenne gala...

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

MJ&M Benefits CureDuchenne and Other Children’s Charities

Thanks to generous donors Mack, Jack & McConaughey shattered their fundraising goal and raised a net total of more than $1.7 million for children in Austin and across the nation. Mack, Jack & McConaughey 2016 was an incredible success...

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Upcoming Events
CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, April 23, Kansas City, MO

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on April 23 in Kansas City, MO for family members and caregivers at the Don Chrisholm Learning Center, 610 E 22nd St., Kansas City, MO from 9 a.m. 2 p.m...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Blingo to CureDuchenne, April 30, 2016, Philadelphia, PA

Join us for Blingo to CureDuchenne on April 30 at 7 p.m. at The Logan in Philadelphia, Center City’s newest hotel. Blingo is a night of celebrity bingo, bling and celebration where Philadelphians come together to help make a huge difference in the lives of those with Duchenne muscular dystrophy...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, May 14, Boston, MA

Please join CureDuchenne for a free, informative class on Duchenne muscular dystrophy on May 14 in Waltham, MA for family members and caregivers at 70 3rd Ave, Waltham, MA from 9 a.m. 2 p.m...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, June 11, Long Island, NY

Please join CureDuchenne for a free, informative class on Duchenne muscular dystrophy on June 11 in Long Island, NY for family members and caregivers at the Long Island Marriott, 101 James Doolittle Blvd., Uniondale, NY from 9 a.m. 2 p.m...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, July 9, Chicago, IL

Please join CureDuchenne for a free, informative class on Duchenne muscular dystrophy on July 9 in Chicago, IL for family members and caregivers at Vitals Rehabilitation, 5820 W Irving Park Rd, Chicago from 9 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

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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