January 2016
Together we can Cure Duchenne!
Research
oct2015_thumb04

CureDuchenne Ventures LLC Provides Funding to Bamboo Therapeutics to Help Advance Gene Therapy Treatment for Duchenne Muscular Dystrophy

CureDuchenne Ventures LLC, a venture philanthropy organization that funds research to find a cure for Duchenne muscular dystrophy, announced today that they have invested in Bamboo Therapeutics, Inc...

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oct2015_thumb04

CureDuchenne Ventures LLC Provides Funding to RASRx to Help Advance Novel Treatment for Duchenne Muscular Dystrophy

CureDuchenne Ventures LLC, a venture philanthropy company that funds research to find a cure for Duchenne muscular dystrophy, announced January 5 that they have invested in RASRx, a start-up company dedicated to developing therapies for rare diseases...

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oct2015_thumb04

PTC Completes Rolling NDA Submission to FDA and Submits Phase 3 ACT DMD Clinical Trial Results to EMA for Translarna™ (ataluren) for Treatment of Nonsense Mutation Duchenne Muscular Dystrophy

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced on January 8 the completion of its rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Translarna™ (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD)...

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Duchenne Research Highlights from MDA Conference

FDA Issues Complete Response Letter for KyndrisaTM for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Marketing Application in Europe Remains Under Review

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced on January 14 that the U.S. Food and Drug Administration (FDA) issued a Complete Response letter to the Company's New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping...

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oct2015_thumb04

CureDuchenne Responds to FDA Decision on Drisapersen for Duchenne Muscular Dystrophy

CureDuchenne, the California-based nonprofit organization dedicated to finding cures for Duchenne muscular dystrophy, released the following statement from its founder and CEO, Debra Miller, following the decision by the Food and Drug Administration (FDA) to send a Complete Response Letter to BioMarin for its exon-skipping drug drisapersen (Kyndrisa)...

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oct2015_thumb04

FDA Postpones Advisory Committee Meeting to Review Eteplirsen Due to Severe Weather Storm in the Washington D.C. Area

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, announced on January 20 that the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Advisory Committee meeting scheduled for Friday, January 22 has been postponed by the FDA due to an anticipated severe winter snowstorm forecasted to hit the Washington D.C. area...

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oct2015_thumb04

CureDuchenne Urges Regulators to Approve Eteplirsen, Stresses the Importance of Multiple Drugs in Pipeline

The FDA released Briefing Document on eteplirsen, the drug for Duchenne muscular dystrophy submitted for approval by Sarepta Therapeutics Inc. Although the review is harsh, we urge the Advisory Committee and the FDA to listen to the patient voice when making a decision...

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oct2015_thumb04

Catabasis Pharmaceuticals Announces Positive Top-Line Results from Part A of the MoveDMD Trial, a Phase ½ Trial of CAT-1004 for the Treatment of Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals, Inc. (NASDAQ: CATB), a clinical-stage drug development company built on a pathway pharmacology technology platform, announced on January 25 positive top-line results from Part A of the MoveDMD trial, a Phase 1/2 trial of CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD or Duchenne)...

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oct2015_thumb14

Dosing and Enrollment in HT-100 Trial Suspended

Dosing and new patient enrollment in all cohorts of the HALO trial, a study evaluating the compound HT-100 in patients with Duchenne muscular dystrophy, a rare disease that results in muscle degeneration and premature death, are being suspended...

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oct2015_thumb14

FibroGen Enrolls First Patients in Phase 2 Clinical Study of Anit-fibrotic Drug Candidate FG-3019 for the Treatment of Duchenne Muscular Dystrophy

FibroGen, Inc. (NASDAQ:FGEN) (“FibroGen”) announced on January 6 enrollment of the first two patients in an open-label, multicenter Phase 2 clinical trial of the investigational compound FG-3019 in patients with Duchenne muscular dystrophy (DMD)...

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oct2015_thumb14

Summit Therapeutics Receives Phase 2 Trial Approval for DMD Patients

Summit Therapeutics PLC (NASDAQ:SMMT) has obtained the approval for the phase 2 proof of concept trial of SMT C1100 candidate, from the UK Medicines and Healthcare products Regulatory Agency (MHRA), and the Research Ethics Committees (RECs)...

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oct2015_thumb14

Gene Editing Offers Hope for Treating Duchenne Muscular Dystrophy, Studies Find

After decades of disappointingly slow progress, researchers have taken a substantial step toward a possible treatment for Duchenne muscular dystrophy with the help of a powerful new gene-editing technique...

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CureDuchenne Cares
Abilities Expo Video Blog

Interest Continues to Rise for Proper Physical Therapy Education within the Duchenne Community

As the Duchenne community anxiously awaits approved treatments, extensive education is crucial to our patients' lives and ability to respond to these treatments as they become available...

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Abilities Expo Video Blog

CureDuchenne Cares Physical Therapist Certification

CureDuchenne Cares Physical Therapist Certification program, a recent addition to the CureDuchenne Cares program, provides additional training to physical therapists on the most current standard of care for working with those who have Duchenne muscular dystrophy...

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

Cheers: Southern California’s Premier Annual Wine Event on February 27

Fine wines. Amazing food. Join us for Southern California’s premier annual wine event Napa in Newport on February 27 at the St. Regis Hotel in Dana Point...

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

Q&A with Debra Miller

CureDuchenne Ventures LLC is a venture philanthropy company that raises funds for Duchenne muscular dystrophy (DMD). It is a for profit branch of the non-profit CureDuchenne Organization.

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

Duchenne and You

PTC announces the launch of a new disease awareness website for the Duchenne community, www.DuchenneandYou.com. The site includes general information about Duchenne and genetics as well as resources for friends and families living with Duchenne...

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

Join The Walk to CureDuchenne

The Walk to CureDuchenne, in partnership with The Paramount Break-A-Leg 5K, will take place on February 14, 2016 at 7:30 a.m. The Walk will not only bring together families, friends, and the Austin community to celebrate the triumphs of the Duchenne community, but will also support the work that CureDuchenne is doing to find a cure...

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

CureDuchenne Cares Workshop for Families, January 30, Austin, Texas

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on January 30 in Austin, Texas for family members and caregivers at the Thompson Center: 2405 Robert Dedman Drive, Austin, TX 78712...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

The Walk to CureDuchenne, February 14, Austin, Texas

Join us for The Walk to CureDuchenne on February 14 in Austin. Create a team, join a team or donate. https://www.crowdrise.com/thewalktocureduchenne...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, February 20, Riverside, California

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on February 20 in Riverside, California for family members and caregivers at the Courtyard Riverside: 1510 University Avenue, Riverside, CA 92507...

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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 40 of Napa's best wineries and world class chef for a truly unforgettable experience. It will be held on February 27, 2016 at the St. Regis Hotel in Dana Point, Calif...

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, March 12, Fort Lauderdale, FL

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy on March 12 in Fort Lauderdale, FL for family members and caregivers at the Embassy Suites Fort Lauderdale, 110 SE 17th Street, Fort Lauderdale, FL 33316.

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CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

CureDuchenne Cares Workshop for Families, Houston, April 9

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

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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