Tag Archives: CureDuchenne

  1. An Open Letter to the Duchenne Community

    Posted on February 13, 201710:39 pm

      CureDuchenne is sharing an expanded and updated open letter to the Duchenne community from Marathon Pharmaceuticals. An Open Letter to the Duchenne Community Dear Members of the Duchenne Community: I want to thank you all for your support and encouragement over the last several years as Marathon worked to bring EMFLAZA™ (deflazacort) through the Read more »

  2. An Open Letter to the Duchenne Community

    Posted on 6:59 pm

      CureDuchenne is sharing an expanded and updated open letter to the Duchenne community from Marathon Pharmaceuticals. An Open Letter to the Duchenne Community  Dear Members of the Duchenne Community: I want to thank you all for your support and encouragement over the last several years as Marathon worked to bring EMFLAZA™ (deflazacort) through the Read more »

  3. FDA Approves EMFLAZA (deflazacort) … Our Experience With Deflazacort

    Posted on February 10, 20174:03 pm

      We are delighted that the U.S. Food and Drug Administration (FDA) has approved EMFLAZA (deflazacort) for the treatment of Duchenne. This is wonderful news for the Duchenne community to have access to this drug in the U.S. We are grateful for Marathon Pharmaceuticals for conducting the multiple studies necessary for FDA approval. When your Read more »

  4. Additional Information about EMFLAZA (deflazacort) for Duchenne Patients under the Age of Five

    Posted on 1:49 am

      Today, the FDA approved the use of EMFLAZA (deflazacort) for Duchenne patients over the age of five years old.  It is routine for the FDA to designate specific patient populations on the “label” that are approved to receive a drug.  The reason the FDA excluded younger patients for EMFLAZA is because there has not Read more »

  5. Physical Therapist Darcy Peach Provides Peace of Mind to Duchenne Families

    Posted on January 26, 20173:51 pm

      Darcy Peach discovered her calling to become a physical therapist while undergoing treatment for a volleyball injury in high school. During her treatment sessions, Darcy’s physical therapist took a great deal of care and time in treating her.  She witnessed first-hand the therapeutic and relationship building benefits that occur during the process of physical Read more »

  6. Deflazacort Transition Program Reminder

    Posted on January 17, 20176:42 pm

      Marathon submitted New Drug Applications (NDAs) to the Food and Drug Administration (FDA) last June for deflazacort. The NDAs have been accepted for filing and review. The FDA expects to complete its review of the applications in early February. If approved, deflazacort will become accessible in the U.S. and it would be the second Read more »

  7. We need your help to advance a treatment to clinical trials for rare, Duplication 2 mutations!

    Posted on December 15, 20166:56 pm

      CureDuchenne has always been focused on finding and funding treatments to help EVERYONE affected with Duchenne, and to TREAT THE WHOLE DISEASE.  We are very proud of our comprehensive portfolio of research projects and we continue to be the research leader for the Duchenne community. Over the past five years we have pioneered duplication Read more »

  8. Pfizer Clinical Trial Update

    Posted on December 8, 20164:33 pm

      CureDuchenne is pleased to share the following updates from Pfizer pertaining to the on-going multicenter Phase II clinical trial of the investigational compound, domagrozumab (PF-06252616) in boys with Duchenne muscular dystrophy (DMD). Domagrozumab is an experimental, infused, anti-myostatin monoclonal antibody.  Myostatin is a naturally occurring protein in muscles that helps control muscle growth; it Read more »

  9. Bonding Through Stretches

    Posted on December 7, 20165:26 pm

      Guest blog by Paul Miller Our son Hawken was diagnosed with Duchenne in the fall of 2002.  Debra and I rolled up our sleeves and began a journey we had not anticipated.   We found there were many variables to managing this disease.  One of these is physical therapy.  Maintaining the health of the muscles Read more »

  10. Take Action for all Those with Duchenne

    Posted on December 5, 20164:45 pm

      As a community, we need to continue our efforts to find a cure for every single person impacted by Duchenne. That means funding research that treats all aspects of the disease.  That includes anti-inflammatory and anti-fibrosis therapies, gene therapy and gene editing; and cardiac therapies that address the leading cause of death for Duchenne.  Read more »

  11. 21st Century Cures Act Passed by House

    Posted on December 1, 201611:03 pm

      Yesterday the House of Representatives passed the draft of the 21st Century Cures Act. It is slated for a vote in the Senate early next week.       The new draft includes: $4.8 billion in new funding for the National Institutes of Health (NIH) $500 million in new funding for the Food and Read more »

  12. With Gratitude this Thanksgiving

    Posted on November 23, 20164:53 pm

      Thanksgiving is more than a chance to appreciate what we have. It’s also about taking the time to appreciate the people who help us in our journey, those who share in our hopes and dreams, and our struggles and our pain. For all of you, we are full of gratitude. We are filled with Read more »

  13. Help Support the 21st Century Cures Act and the OPEN ACT

    Posted on November 17, 20164:56 pm

      The final decisions could be made today on the 21st Century Cures Act and the OPEN ACT – a provision that could be transformational for rare disease patients. CureDuchenne signed the letter of support. Despite outreach from constituents, we are still hearing pushback from Sen. Warren on the OPEN ACT. If you could please Read more »

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

    Posted on November 16, 20166:36 pm

      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. In Read more »

  15. Marathon Webinar: Update on Deflazacort Access and Regulatory Status

    Posted on November 4, 20164:48 pm

    Thank you to Marathon Pharmaceuticals for providing updated information about deflazacort access and regulatory status during a webinar this week.  The webinar addressed Marathon’s Expanded Access Program for deflazacort and what a potential FDA approval of deflazacort would mean in terms of access to the drug in the U.S.  The webinar has been archived.  https://cc.readytalk.com/play?id=hl12zw. Read more »

  16. Better Designed Clinical Trials Now

    Posted on October 31, 20168:25 pm

      As a parent of a son with Duchenne I have been frustrated that the data presented to the FDA does not match the patient experience.  I personally know so many patients who have been in recent clinical trials that are doing much better than expected and are seeing a slower progression of their disease Read more »

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

    Posted on October 12, 20164:28 pm

      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.  It is these multiple roles and complicated diseases that keep Jerry Hsu, an Orange County, California-based physical therapist, excited about his job every day. Read more »

  18. Learn. Engage. Empower.

    Posted on October 10, 20169:15 pm

      Caring for a child with Duchenne can be daunting, complicated, and often times, isolating. But it doesn’t have to be. On October 22, CureDuchenne Cares will hold its inaugural Family Summit bringing together Duchenne families, medical providers and researchers for a daylong event focused on education and fellowship. The goal of this summit is Read more »

  19. We are “All in Building a Bridge over Duchenne”

    Posted on September 21, 20164:02 pm

      Guest blog by Tammy and Scott Henegar Our family’s journey with Duchenne muscular dystrophy started in March 2011 when our 4 ½ year old adventurous and sweet young boy was diagnosed with the heart breaking diagnosis that no parent should have to face.  We were told that Braedan would be facing a life of Read more »

  20. First Drug Approved for Duchenne: A Beacon of Hope for the Duchenne Community

    Posted on September 20, 20165:08 pm

      My husband Paul and I have dreamed of this day since our son Hawken was diagnosed with Duchenne almost 15 years ago! We are overwhelmed with joy to announce that the FDA has officially granted accelerated approval for Eteplirsen for the treatment of Duchenne Muscular Dystrophy. This is the first approved drug-therapy and a Read more »

  21. Our Journey Dealing With Duchenne

    Posted on September 8, 20167:31 pm

      Guest blog by Maribel and Ramiro Munoz Our son Joshua was born in 2007. Joshua, our oldest son, is an amazing and fun-loving boy. He was diagnosed with Duchenne Muscular Dystrophy in 2013. Duchenne is a progressive muscle degenerative disease that impacts 1 in every 3,500 boys. It starts by limiting their ability to play and run in Read more »

  22. Navigating College, Living with Duchenne

    Posted on August 16, 20167:19 pm

      Guest blog by Hawken Miller For those with Duchenne Muscular Dystrophy the experience and education of a four-year university is likely within your grasp, and being away from home is a definite possibility. I’ll admit, I had my own second thoughts about going off to college, but it is worth it once you move Read more »

  23. Pfizer, Bamboo and CureDuchenne – An Endorsement on the Right Track

    Posted on August 1, 20167:24 pm

      Today, we are very pleased to announce that Pfizer has acquired Bamboo Therapeutics, a gene therapy company we invested in earlier this year through CureDuchenne Ventures. The deal is valued at up to $645 million. Bamboo, led by Dr. Richard Jude Samulski, PhD, the Director of the Gene Therapy Center at UNC, and Dr. Xiao Read more »

  24. Summer Road Trip

    Posted on July 13, 20164:49 pm

      Guest blog by Hawken Miller Many of the things I don’t think are possible for me to do really are. Once I get over my initial apprehension and thoughts about what I won’t be able to do and I open my mind I find that anything I set my mind to is possible. This Read more »

  25. BioMarin Update for the Duchenne Community: 7th July, 2016

    Posted on July 7, 20163:00 pm

      BioMarin is providing a further update to the Duchenne Community following our announcement of the withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on compounds BMN 044, BMN 045 and BMN 053. Update for Patients Receiving Drisapersen, BMN 044, 045 or 053 in Clinical Read more »