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November 2016
CureDuchenne Newsletter - Together We Can Cure Duchenne!
Research
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CHMP Recommends Renewal of Translarna's™ Marketing Authorization for Nonsense Mutation Duchenne Muscular Dystrophy Based on Continued Positive Benefit-Risk Assessment

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced November 11 that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients five years and older...

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Capricor Therapeutics Announces Plans to Expand Clinical Development Program in Duchenne Muscular Dystrophy to Evaluate Peripheral and Respiratory Muscle Function

Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other serious medical conditions, announced on November 10 that it intends to expand its CAP-1002 clinical development program in Duchenne muscular dystrophy (DMD) to encompass the skeletal muscle aspects of the disease, in addition to the cardiac complications...

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Summit Enrolls Patients in the US into PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid

Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') and Clostridium difficile infection, on November 16 announces that it has enrolled its first patients at trial sites in the US into PhaseOut DMD, a Phase 2 proof of concept clinical trial of ezutromid in patients with DMD. Ezutromid dosing is expected to follow within a screening period of up to 28 days...

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Cureduchenne Cares
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CHLA Cares Workshop

On Saturday, November 5, CureDuchenne Cares held its final Family Workshop of 2016. The successful workshop was hosted by Children’s Hospital Los Angeles, which was a welcome collaboration between CureDuchenne and the CHLA Neuromuscular Clinic led by Clinic Director/Neurologist Dr. Leigh Ramos-Platt...

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Video on Multiple Disciplinary Care Model

Dr. Leigh Ramos-Platt from Children's Hospital Los Angeles recently presented during the CureDuchenne Cares Family Summit on the multiple disciplinary care model. Watch the video to learn more...

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Oral Health Consideration Video

Those with Duchenne muscular dystrophy may be at increased risk for oral health complications. This video with Dr. Parish Sedghizadeh of USC School of Dentistry helps to explain some of the conditions, causes and symptoms to be aware of...

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Spotlight on Sandi Preston, PT, CureDuchenne Cares Certified Physical Therapist

Understanding a patient’s motivation for treatment is the key for a physical therapist to empower that patient for the best outcome. For a patient with Duchenne muscular dystrophy, a terminal disease that deteriorates muscle tissue, this empowerment can be the difference between more cherished ambulatory time or losing their drive for therapy altogether...

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

Chef Amar Santana Comes on as the Celebrity Chef for the Third Annual Napa in Newport

Chef Amar Santana of the celebrated Broadway by Amar Santana and Vaca restaurants will be the celebrity chef for the much anticipated third annual Napa in Newport Wine Auction. Chef Amar Santana is one of Orange County’s hottest rising stars who is undeniably transforming the OC culinary scene into a food destination...

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

Webinar: Update on Deflazacort Access and Regulatory Status

CureDuchenne was pleased to host a webinar with Marathon Pharmaceuticals on November 1. Marathon provided updated information about deflazacort access and regulatory status...

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

cTAP Announces Two Research Publications Categorizing and Predicting Disease Progression in Duchenne Muscular Dystrophy

The Collaborative Trajectory Analysis Project (cTAP), a public-private partnership to accelerate data science solutions to critical problems in drug development for Duchenne Muscular Dystrophy (DMD), today announced the publication of two research studies with important implications for the design of effective clinical trials...

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

Give While You Shop

Give a percent of your Amazon purchase to CureDuchenne this holiday season by making your purchases on Amazon Smile. Amazon Smile donates 0.5% of all qualifying purchases to the charity chosen by buyer. Millions of product qualify, and will be indicated on the item pages...

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

CBS Courage in Sports: Climb to CureDuchenne

Mountain climber Tyler Armstrong was featured in CBS Courage in Sports showcasing how he climbs mountains to raise awareness and fund research to help find a cure for Duchenne muscular dystrophy...

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Upcoming Events
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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 30 of Napa’s most distinguished wineries to Orange County for an extraordinary wine and culinary experience...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Champions to CureDuchenne, April 8, 2017, Austin, TX

Save the date for the Champions to CureDuchenne gala on April 8 at the University of Texas Golf Club. Join us for a magical evening to find a cure for Duchenne muscular dystrophy. The event includes cocktails, dinner, casino, live and silent auction...

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