March 2016
Together we can Cure Duchenne!
Research
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FDA sets new date for hearing on Sarepta’s Duchenne drug

Nearly two months after the Food and Drug Administration postponed what would have been one of the most well-attended advisory committee hearings ever for a drug, this morning the agency announced it has rescheduled the hearing on a drug to treat Duchenne muscular dystrophy by Sarepta Therapeutics...

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Catabasis Pharmaceuticals Presents Positive CAT-1004 Data from Part A of the MoveDMDSM Trial at the 2016 Muscular Dystrophy Association Clinical Conference

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced March 21 positive data from Part A of the MoveDMD trial of CAT-1004 for Duchenne muscular dystrophy (DMD)...

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UF Powell Gene Therapy Center is site for Duchenne Muscular Dystrophy clinical trial

Researchers at the University of Florida Powell Gene Therapy Center are part of an innovative clinical trial aimed at repairing hearts damaged by the effects of Duchenne muscular dystrophy...

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Sarepta 4-Year Data Analysis Of Eteplirsen To Treat DMD

The FDA has discounted the External Controls submitted by Sarepta. Here, we establish that intermittent vs. continuous dosing of steroids is insignificant. Furthermore, we demonstrate that P values for the 6MWT comparison between treatments with Eteplirsen vs. External Controls have increased consistently culminating in significant values for years three and four...

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Duchenne Research Highlights from MDA Conference

PTC Therapeutics Provides Update on Health Canada Review of Translarna™ (ataluren) for the Treatment of Duchenne Muscular Dystrophy

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced on March 14 that the company intends to submit the results of the recently completed Phase 3 ACT DMD study for review by Health Canada as part of the New Drug Submission (NDS) for Translarna™ (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD)...

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Duchenne Research Highlights from MDA Conference

Marathon Pharmaceuticals Announces Nationwide Expanded Access Program for Patients with Duchenne Muscular Dystrophy (DMD)

Marathon Pharmaceuticals, a leader in developing treatments for rare diseases, announced on March 9 that it is expanding patient access to deflazacort, its investigational medicine for Duchenne Muscular Dystrophy (DMD)...

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Duchenne Research Highlights from MDA Conference

Dog with Duchenne Muscular Dystrophy Escapes the Disease, Spurring Interest in Potential New Treatment Approach

A golden retriever carrying the gene mutation for Duchenne muscular dystrophy (DMD) unexpectedly remained healthy for life, sparking interest in a new avenue of investigation into DMD treatment...

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Duchenne Research Highlights from MDA Conference

PTC Therapeutics Patient/Parent Call

Stu Peltz from PTC Therapeutics hosted a call on March 1 regarding the Refuse to File letter from the FDA, dated February 22nd. More detailed information is available in the transcript from the earnings call on February 29...

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CureDuchenne Cares
Abilities Expo Video Blog

CureDuchenne Cares is Giving our Sons with Duchenne the Best Care Possible

While CureDuchenne is filling the therapeutic drug pipeline, CureDuchenne Cares is dedicated to bringing the same level of care that Debra and Paul Miller’s son, Hawken, receives to all boys across the country...

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

Seventh Annual Champions to CureDuchenne Gala Presented by Johnny Carino’s To Be Held April 9 at The University of Texas Golf Club

Champions can come in all sizes and shapes, but none have bigger hearts than Texans who are fighting to find a cure for Duchenne muscular dystrophy. On April 9, hundreds will gather at The University of Texas Golf Club for the seventh annual Champions to CureDuchenne gala to raise awareness and funds to help CureDuchenne in its mission to find a cure for this deadly genetic disease....

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

CureDuchenne a Beneficiary of MJ&M

Once again CureDuchenne will be a beneficiary of Mack, Jack & McConaughey (MJ&M), a joint fundraising effort of Academy Award-winning actor Matthew McConaughey, ACM Award-winning recording artist Jack Ingram and ESPN analyst and Texas coaching legend Mack Brown...

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

Bring on the Blingo

Join us for a night of celebrity bingo and bling at the third annual Blingo to CureDuchenne on April 30 at the Logan in Philadelphia. Blingo brings notable VIPs, business and community leaders of Philadelphia to make a difference for thousands of children and young adults who suffer from Duchenne muscular dystrophy...

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

CureDuchenne Heroes Video

CureDuchenne is funding lifesaving research for children with Duchenne muscular dystrophy. You can help contribute to medical history. You can help transform a terrible disease...

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

NORD Announces 2016 Rare Impact Award Recipients to Be Honored on May 17

Continuing its annual tradition of recognizing the people, organizations, advocates and companies who are making a difference in the fight against rare diseases, the National Organization for Rare Disorders (NORD) announces the 2016 Rare Impact Award recipients to be honored on May 17...

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

Napa In Newport Raises More Than $700,000 To Help Find a Cure for Duchenne Muscular Dystrophy

More than 420 guests gathered at the St. Regis on Saturday, Feb. 27 to enjoy the fruits of 41 top Napa Valley wineries and find a cure for Duchenne Muscular Dystrophy. The second annual Napa in Newport wine auction raised more than $700,000 to support CureDuchenne’s mission to find a cure for this genetic disease that causes muscle degeneration...

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Upcoming Events
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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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, April 9, Houston, TX

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on April 9 in Houston, TX for family members and caregivers at the T.E.A.M Approach: 13150 FM 529, Suite 114, Houston, TX 77041...

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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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