The 2017 American Society of Gene & Cell Therapy took place in Washington DC last week. The Duchenne community was treated to a wealth of data from projects utilizing gene editing and gene therapy to treat Duchenne muscular dystrophy. On Wednesday, May 10, the well-attended session entitled “Gene Therapies for Musculoskeletal Diseases was chaired by […]
PTC is committed to getting patients onto EMFLAZA as quickly as possible, without interruption of therapy. For patients that are currently accessing EMFLAZA through Master Pharmacy importation from outside of the US, they may have immediate access to a “Bridge Program” that will provide EMFLAZA free of charge while their insurance status is being investigated. […]
Twenty years ago I was honored with the greatest gift I could have ever imagined, motherhood. As I think about the most precious gift in my life, my son Hawken, I am elated with happiness. Watching him grow up, his curiosity, big heart and sense of humor continue to inspire me and give me hope […]
Today, Dr. Eric Olson, PhD, Chair of Molecular Biology at UT Southwestern, was honored to give a presentation at the American Society of Gene & Cell Therapy (ASGCT) 2017 meeting in Washington DC for the George Stamatoyannopoulos Lecture series. Dr. Olson presented new research showing how gene editing technologies like CRISPR/Cas9 and Cpf-1 can be […]
At CureDuchenne we understand that when families are affected by a disease with no cure such as Duchenne muscular dystrophy, finding a sense of community is important. We are pleased to introduce our new CureDuchenne website, which is focused on strengthening the Duchenne community and connecting families to the best resources available. Our updated website […]
Today, PTC Therapeutics hosted a call for the Duchenne community to announce pricing and services for their launch of EMFLAZA, the commercial name for deflazacort.  The announced price will be  $35,000/year for a 25kg patient. This is the net pricing to payors.  PTC is committed to provide access and availability of EMFLAZA at no or […]
Mother’s Day is a reminder to love our moms a little extra and be thankful for all that they do for us. For boys with Duchenne, it’s a reminder of how their mothers impact their lives in the largest ways and with the smallest gestures. The boys say it best. Here’s some of their stories: […]
May 8, 2017   Dear Duchenne Families, Today we shared our plans for the launch of EMFLAZA™ (deflazacort), the first FDA-approved corticosteroid for Duchenne muscular dystrophy patients age 5 and older, regardless of the genetic mutation. We are excited to launch EMFLAZA in the U.S., and we will begin making the product commercially available to […]
CureDuchenne is pleased to see the positive preliminary data from Capricor Therapeutics on their HOPE clinical trial in Duchenne muscular dystrophy. CureDuchenne Ventures helped fund this clinical trial to address cardiac failure, the primary cause of death for Duchenne patients.  This data provides hope to  families with Duchenne. Capricor Therapeutics announced positive top-line results from […]
April 20, 2017   Dear Duchenne Community, Today we announced that we have completed our acquisition of Emflaza™ (deflazacort), the first FDA-approved, anti-inflammatory therapy for Duchenne Muscular Dystrophy (DMD) patients age 5 and older regardless of the genetic mutation. We are pleased the acquisition completed ahead of schedule, following early conclusion of the antitrust review […]
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