October 2016
Together we can Cure Duchenne!
Research
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Nonprofits, Pharmaceutical Companies and Noted Clinicians Join to Launch the Collaborative Trajectory Analysis Project (cTAP), Advancing Clinical Trial Design for Duchenne Muscular Dystrophy

The next wave in tackling Duchenne Muscular Dystrophy is getting a boost from a collaboration of nonprofit organizations, biopharmaceutical companies and noted clinicians who have joined together to advance the progress of drug development and bring quality of life treatments to the Duchenne community sooner...

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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) announced on October 15 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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Catabasis Pharmaceuticals Completes Target Enrollment for Part B of the MoveDMDŽ Trial, a Phase 2 Trial of Edasalonexent (CAT-1004) for the Potential Treatment of Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals, Inc. (NASDAQ: CATB), a clinical-stage pharmaceutical company, announced on October 4 that target enrollment of 30 patients has been reached for Part B of the MoveDMD trial, a 12-week trial to assess the safety and efficacy of edasalonexent (CAT-1004) in Duchenne muscular dystrophy (DMD)...

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Sarepta Therapeutics and Summit Enter Into Exclusive License and Collaboration Agreement for European Rights to Summit’s Utrophin Modulator Pipeline for the Treatment of Duchenne Muscular Dystrophy

Sarepta Therapeutics (NASDAQ: SRPT) and Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM) announced October 26 that they have entered into an exclusive license and collaboration agreement granting Sarepta rights in Europe, as well as in Turkey and the Commonwealth of Independent States (‘the licensed territory’), to Summit’s utrophin modulator pipeline, including its lead clinical candidate, ezutromid, for the treatment of Duchenne muscular dystrophy (‘DMD’)...

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PTC Therapeutics Announces New Analyses that Support the Potential Benefit of Ataluren in Preserving Lung Function in Non-Ambulatory Nonsense Mutation Duchenne Muscular Dystrophy Patients

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced on October 6 new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory nonsense mutation Duchenne muscular dystrophy patients (nmDMD)...

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PTC Therapeutics Provides Regulatory Update on Translarna™ (ataluren) for Nonsense Mutation Duchenne Muscular Dystrophy

PTC Therapeutics, Inc. (NASDAQ: PTCT) on October 17 provided a regulatory update on Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD)...

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CureDuchenne Cares
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CureDuchenne Cares Hosts Inaugural Family Summit This October

On Saturday, October 22, 2016, CureDuchenne Cares held its inaugural Family Summit in Garden Grove, CA...

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Spotlight on Jerry Hsu, PT, CureDuchenne Cares Certified Physical Therapist

A physical therapist is required to take on multiple roles while caring for their patients, and this is especially true when treating a complex condition such as Duchenne muscular dystrophy...

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

RARECast: A Controversial Approval for a Duchenne Drug and What Lies Ahead

Listen in to this podcast with Debra Miller on the significance of the approval, CureDuchenne's venture philanthropy model, and what other hope the Duchenne drug pipeline may hold for patients...

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

Bamboo Therapeutics Featured on Worldwide Business with Kathy Ireland

CureDuchenne funded Bamboo's gene therapy research. Bamboo has since been acquired by Pfizer...

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

Cambridge Turns Vintage Vegas During Dealing for Duchenne

More than 300 guests attended Dealing for Duchenne Cambridge on October 20 to celebrate the first FDA approved treatment for Duchenne and to provide hope for all those living with Duchenne...

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

All in for Duchenne Builds Bridges to Find Treatments for Duchenne

Braedan’s Bridge hosted their 4th Annual “All in for Duchenne” Dinner/Reverse Raffle on October 15 and it was bigger and more exciting than ever...

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

Dealing for Duchenne San Antonio Hits the Jackpot

The San Antonio community came together on October 15 at Pedrotti’s North Wind Ranch in Helotes, Texas for a night a hope to help find a cure for Duchenne muscular dystrophy...

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Upcoming Events
CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Webinar with Marathon, November 1

CureDuchenne is hosting a webinar with Marathon on November 1, 2016 at 2:00 p.m. ET/11:00 a.m. PT for a webinar update about matters relating to deflazacort...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, November 5, Los Angeles, CA

Please join CureDuchenne for a free, informative class on muscular dystrophy on November 5 in Los Angeles for family members and caregivers at Children’s Hospital Los Angeles, 4650 W Sunset Blvd., Los Angeles, CA 90027 from 8:30 a.m. to 2 p.m...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

Napa In Newport, March 4, 2017, Laguna Niguel, 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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