December 2015
Together we can Cure Duchenne!
Letter from Founder
FDA Advisory Committee Discusses Clinical Data Package for BioMarin's Kyndrisa(TM) (drisapersen) for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Letter from Founder

It’s been a landmark year for our family as well as CureDuchenne. Advances in Duchenne research, new care initiatives and our remarkable son, Hawken, have given us much to be thankful for. Your continued kindness and support has allowed CureDuchenne to further our mission to save the lives of those with Duchenne...

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Year End
Sarepta Therapeutics Announces Publication of Positive Long-Term Safety and Efficacy Data for Eteplirsen in the Annals of Neurology

A Father’s Dream for his Sons

When I was 5 years old, I dreamed of becoming a doctor. My mom had just lost her battle with breast cancer and I wanted to find a cure so that no other children would ever feel that same loss...

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BioMarin Announces Data Analysis Demonstrating Consistent Efficacy of Kyndrisa™ (drisapersen) in Comparable Patients Across Three Randomized Studies

Grandfather’s Thoughts about Duchenne

I am the grandfather of two little boys with Duchenne, Timothy and Andrew. Nothing means more to me than to have them hug my neck and to give them hugs...

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Research
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BioMarin Announces That FDA Has Advised it Will Not Take Action on the Kyndrisa™ (drisapersen) New Drug Application by the PDUFA Date

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced that the U.S. Food and Drug Administration (FDA) has notified the Company that they had not yet completed their review process and would be unable to take an action by the Prescription Drug User Fee Act (PDUFA) action date for KyndrisaTM (drisapersen) of December 27, 2015, and anticipate taking action in early January 2016...

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oct2015_thumb04

Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, announced on December 18 that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Sarepta’s New Drug Application (NDA) for eteplirsen, on January 22, 2016...

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oct2015_thumb04

Catabasis Pharmaceuticals Completes Enrollment for Part A of the MoveDMDSM 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, today announced that enrollment is complete in Part A of the MoveDMD trial, a Phase 1/2 trial of CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD)...

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Duchenne Research Highlights from MDA Conference

Marathon Pharmaceuticals Launches an Expanded Access Program to Provide Deflazacort to U.S. Patients with Duchenne Muscular Dystrophy

Marathon Pharmaceuticals, a biopharmaceutical company that develops treatments for rare neurological diseases, today announced that, under the authorization of the U.S. Food and Drug Administration (FDA), it will provide deflazacort to qualified patients with Duchenne muscular dystrophy (DMD) through an open-label, expanded access program (EAP) called ACCESS DMD™...

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oct2015_thumb04

New Clinical Study for DMD Patients

This study is focused on the development of an investigational anti-myostatin adnectin drug (BMS-986089) for Duchenne Muscular Dystrophy. The study is designed to evaluate the safety and tolerability of this new investigational drug in ambulatory boys with Duchenne...

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oct2015_thumb04

UF Researchers Announce Gene Therapy Project for Duchenne Muscular Dystrophy

The University of Florida’s Powell Gene Therapy Center has partnered with a biotechnology company to launch a gene therapy project aimed at developing potential treatments for Duchenne muscular dystrophy, the most common form of muscular dystrophy...

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oct2015_thumb04

Elusive Sources of Variability of Dystrophin Rescue by Exon Skipping

Systemic delivery of anti-sense oligonucleotides to Duchenne muscular dystrophy (DMD) patients to induce de novo dystrophin protein expression in muscle (exon skipping) is a promising therapy...

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oct2015_thumb14

SickKids First to Remove Duplicated Gene Using CRISPR

It’s not often that researchers around the world collectively tout a technology as truly transformational. CRISPR is being called a game changer. It is a gene editing tool more precise, efficient and cheaper than its predecessors, and it is changing the way scientists and clinicians approach the treatment of genetic disorders...

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CureDuchenne Cares
Abilities Expo Video Blog

New CureDuchenne Cares Video

Standard of care for those with Duchenne muscular dystrophy is critical. It is important to keep those with Duchenne as healthy as possible as long as possible so they can benefit from approved treatments when they become available...

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Abilities Expo Video Blog

Massage Program for Duchenne

Massage is a great addition to the regular home program for those with Duchenne muscular dystrophy...

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

Local Boy Fights FDA for Drug

Damon Woods from San Diego shares his story of waiting for an approved drug from the FDA. Watch the news segment from NBC 7 in San Diego...

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

(SOS) Sign To Save Our Sons, give our Duchenne boys their Christmas miracle. FDA Approve Drisapersen

My name is Tonya Carlone, mother of Gavin who has Duchenne Muscular Dystrophy.  This is a rare FATAL muscle weakening disease.  Typically boys with Duchenne lose their ability to walk by age 12 and are confined to a wheelchair...

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

Family Awaits Decision from FDA about Life-altering Drug for Son

A Walker County family is trying to bring national attention to a disease that commonly affects young boys...

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

Renowned Wineries Team up for the Second Annual Napa in Newport on Feb. 27 to Save the Lives of Boys with Duchenne Muscular Dystrophy

The date for the highly anticipated 2016 Napa in Newport has been set for February 27 at the St. Regis Hotel in Dana Point. Southern California’s premier annual wine event, Napa in Newport unites 40 renowned vintners and wine enthusiasts to help find a cure for Duchenne muscular dystrophy, a devastating genetic disease that robs children and young adults of their muscle strength and claims their lives by their mid-20s...

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

Give a Percent of Your Amazon Purchase to CureDuchenne

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...

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Upcoming Events
CureDuchenne Venture Philanthropy Model Featured in Forbes Insight Report

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

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 as many as 40 of Napa's best wineries and world class chef for a truly unforgettable experience...

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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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