Tag Archives: muscular dystrophy

  1. An Open Letter to the Duchenne Community

    Posted on February 13, 20176:59 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 »

  2. 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 »

  3. 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 »

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

    Posted on January 18, 20168:17 pm

      The Duchenne community continues its fight to get an approved treatment for Duchenne. 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 Read more »

  5. BioMarin DMD Update for Patient Groups

    Posted on January 15, 20167:02 pm

      We wanted to share the following information from BioMarin in regards to yesterday’s news on the FDA decision on Kyndrisa (drisapersen).     Dear Patient Group Representative, As you are likely aware, yesterday, we announced that the FDA issued a Complete Response Letter to BioMarin for our new drug application for Kyndrisa (drisapersen) for Read more »

  6. CureDuchenne-Funded Trial for Cardiac Therapy

    Posted on January 13, 201611:54 pm

      Capricor Therapeutics is actively recruiting participants for a clinical trial for a potential cardiac cell therapy for those with Duchenne muscular dystrophy.  CureDuchenne funded Capricor to support this trial that addresses heart disease, which is the primary cause of death for those with Duchenne.     The Halt cardiOmyopathy ProgrEssion in Duchenne (HOPE-Duchenne) is a Read more »

  7. Cardiac Trial for Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy

    Posted on November 5, 20154:51 pm

      Did you know that heart failure is very common and often fatal in patients with Duchenne muscular dystrophy? The Halt cardiOmyopathy ProgrEssion in Duchenne (HOPE-Duchenne) is a clinical trial that will evaluate whether an investigational cardiac cell therapy is safe and potentially effective in minimizing scar and improving heart function in DMD associated heart Read more »

  8. Transfer Techniques to Protect Duchenne Patients and Caregivers

    Posted on November 3, 20156:13 pm

      Moving someone who has Duchenne from one location to another is no easy task, especially when considering patient and caregiver safety. Physical therapist,  owner at Duchenne Therapy Network and CureDuchenne Cares program director, Jennifer Wallace approaches this topic in her recent blog video highlighting the importance of using proper techniques when performing transfers. Transfers Read more »

  9. Showing Nepal’s Duchenne Community our Southern California Hospitality

    Posted on October 23, 20158:12 pm

      To turn away from any member of the global Duchenne community would be to turn away from our own here at home. That’s why we at CureDuchenne, the non-profit that’s dedicated to finding a cure, seek out and support Duchenne communities around the world. We are honored to provide much needed support for those Read more »

  10. Speak Up for the Duchenne Community!

    Posted on October 14, 201511:18 pm

      After waiting for a very long time, the Duchenne community now has clarity on the Advisory Committee meetings dates for BioMarin and Sarepta.  BioMarin’s date is November 24, 2015 and Sarepta’s date is January 22, 2016.  Please remember, these dates are tentative until they actually appear on the Federal Registry.  Both of the Ad Read more »

  11. Expanding and Improving Duchenne Standard of Care

    Posted on September 23, 20154:01 pm

      As a mom of a son with Duchenne, I understand the importance of physical therapy in this disease.  Early after Hawken’s diagnosis, we were referred to a physical therapy facility that knew very little about the nuances of physical therapy for Duchenne.  The PT’s in that state supported clinic actually used strength building exercises Read more »

  12. Welcome Katie Mastro

    Posted on September 22, 20153:59 pm

      We are delighted to announce that Katie Mastro has joined joined CureDuchenne as program services manager. She is responsible for managing and expanding the CureDuchenne Cares program, engaging with families whose loved ones have Duchenne Muscular Dystrophy, and coordinating events. Katie jumped right in and participated in this past weekend’s CureDuchenne Cares workshop in Read more »

  13. Researchers Create New Mouse Model for Duchenne with Duplication of Exon 2

    Posted on September 16, 20159:58 pm

      Dr. Kevin Flanigan and his team at the Flanigan Lab at Nationwide Children’s Hospital are hard at work researching duplication mutations. They have created a new mouse model for Duchenne muscular dystrophy with a duplication of exon 2. Their research was recently published in Neuromuscular Disorders (http://www.ncbi.nlm.nih.gov/pubmed/26365037). “CureDuchenne funded our research early on and Read more »

  14. Welcome Dr. Jak Knowles

    Posted on 3:46 pm

    We couldn’t be prouder that CureDuchenne has played a critical role in the development of what we hope will be the first-ever pharmaceutical treatments for Duchenne muscular dystrophy. Investments by CureDuchenne in Prosensa (now BioMarin) and Sarepta Therapeutics have helped advance two drugs through clinical trials that await FDA approval in the fall of 2015. Read more »

  15. Passion Meets Courage

    Posted on September 9, 20153:49 pm

      To say I have witnessed young men and boys with Duchenne face heartbreaking challenges is an understatement. Duchenne is one of the toughest diseases in the world. That’s why when I hear words like courage, I immediately think of our boys with Duchenne. Today I wanted to reflect on very courageous young boy who Read more »

  16. Do You kNOW Your Duchenne?

    Posted on September 2, 20154:35 pm

      When our son, Hawken was diagnosed with Duchenne almost 13 years ago, my husband and I felt there couldn’t be too many things worse for a family to experience.  In the years since, I’ve met hundreds of Duchenne families and I’ve realized that many parents had a worse experience than we did. Hawken was Read more »

  17. FDA Webinar on Accelerated Approval Summary

    Posted on February 21, 20131:28 am

        CureDuchenne was honored to host a webinar today, in collaboration with PPMD and MDA to discuss accelerated approval for drugs for rare diseases.   Dr. Robert Temple, director of the Office of Medical Policy of FDA’s Center for Drug Evaluation and Research presented detailed information on the different options available to speed the approval of Read more »