This summer, PPMD will host a pre-conference the day before the , addressing the issue of transitioning individuals living with Duchenne from pediatrics, through adolescence and into adulthood. We realize that this is a very complex process that will require a program that should begin as early as age 12, and will extend well into adulthood. The purpose of the pre-conference is to provide a first step that will bring together parents, patients, providers, and policy makers to begin to…Continue
Added by Kathi Kinnett on May 10, 2013 at 11:41am — No Comments
Last June, just prior to the Connect Conference, PPMD hosted the first Transforming Duchenne Care meeting. This meeting was attended by health care providers and administrators from 17 institutions, parents, and representatives from MDA, CDC, NIH, and industry. Key themes emerged from this meeting:
Added by Kathi Kinnett on May 3, 2013 at 10:00am — No Comments
Left ventricular assist devices (LVADs) are fairly new to the pediatric population and are very new to the Duchenne community. So new, in fact, that, in the fall of 2012, only three patients have had LVADs implanted worldwide. As dilated cardiomyopathy and heart failure remain the major cause of death in patients with Duchenne, this novel therapy could possibly have far reaching effects. Below are two LVAD stories, from two different perspectives: first a young man with Duchenne (Jason)…Continue
Added by Kathi Kinnett on May 1, 2013 at 12:30pm — No Comments
In June 2012, PPMD sponsored the first Transforming Duchenne Care meeting. The meeting was organized in response to clinical care discrepancies that exist between centers treating patients with Duchenne/Becker muscular dystrophy in North America. The purpose of this…Continue
Emergencies always happen when you are away from home, after “office hours,” or over a holiday. Due to this helpful timetable, it is likely that, when an emergency occurs, patients will be seen by professionals who have no knowledge of them or of their (rare) disease. During a respiratory emergency, the first reaction of emergency personnel is to give oxygen. Giving oxygen, without proper monitoring, can be life threatening to a person with Duchenne. Several such emergencies happened this…Continue
Added by Kathi Kinnett on February 12, 2013 at 11:00am — No Comments
On January 24, PPMD hosted the third in our series of four cardiac webinars. This webinar was dedicated to the subject of Ventricular assist device (VAD) use in patients with Duchenne. We were pleased to be joined by a panel of four outstanding surgeons who are all experts in the field of cardiothoracic surgery.
Added by Kathi Kinnett on January 30, 2013 at 12:30pm — No Comments
End stage dilated cardiomyopathy (DCM) is a known complication of Duchenne muscular dystrophy. DCM leads to end stage heart failure and the premature death of patients with Duchenne. Given recent advances in respiratory care, heart failure is the cause of death in more than 40% of patients with Duchenne.
A “ventricular assist device (VAD)” is a mechanical pump that is connected to the heart in order to support its function. The type of device that would be considered in…Continue
Added by Kathi Kinnett on January 16, 2013 at 1:30pm — No Comments
The 194th workshop of the European Neuro Muscular Centre (ENMC) met in Naarden, The Netherlands, December 7-9, 2012. The title of the workshop was, “Towards Clinical Application of Antisense-mediated Exon Skipping for Duchenne Muscular Dystrophy – Opportunities and Challenges.” 25 representatives of industry, research, care and advocacy from around the world…Continue
Added by Kathi Kinnett on December 13, 2012 at 12:30pm — No Comments
Parent Project Muscular Dystrophy recently hosted the second part of our Cardiac Webinar Seriers with Dr. Larry Markham (Children’s Hospital Vanderbilt, Vanderbilt, TN) and Dr. J. Lynn Jefferies (Cincinnati Children's Hosptial, Cincinnati, OH) to discuss cardiac…Continue
Added by Kathi Kinnett on December 5, 2012 at 4:30pm — No Comments
It’s been a little while since our last update, but I wanted to wait until I had definite news. It’s been an exciting month for Transforming Duchenne Care (TDC), and I will do my best to get everyone up to speed. We had two important and informative meetings. Below is an update on our recent meeting with the Cystic Fibrosis Foundation, as well as PPMD’s contribution to Duchenne care.…Continue
Added by Kathi Kinnett on November 15, 2012 at 12:00pm — No Comments
It’s been a little while since our last update, but I wanted to wait until I had definite news. It’s been an exciting month for Transforming Duchenne Care (TDC), and I will do my best to get everyone up to speed. We had two important and informative meetings. Below is an update on our recent meeting with the MDA. Click here to read about our…Continue
Added by Kathi Kinnett on November 15, 2012 at 11:30am — No Comments
In light of PPMD’s Cardiac Webinar Series, our Director of Care, Kathi Kinnett answers questions/concerns about Ventricular Assist Devices (VAD).
Added by Kathi Kinnett on October 11, 2012 at 2:30pm — No Comments
Parent Project Muscular Dystrophy recently hosted the first part of our Cardiac Webinar Seriers with Dr. Linda Cripe (Nationwide Children’s Hospital, Columbus, OH) and Dr. Kan Hor (Cincinnati Children's Hosptial, Cincinnati, OH) to discuss monitoring heart disease in Duchenne and carrier moms/daughters.
Added by Kathi Kinnett on October 9, 2012 at 3:30pm — No Comments
“Lauren’s Hope” started out as “You Name It,” and began as a fun little business making personalized gifts for women and children. They were approached by Lauren’s Hope for A Cure to…Continue
Cardiac care and cardiomyopathy remain major areas of interest and concern for all people with Duchenne. While advances in respiratory care have improved respiratory outcomes, dilated cardiomyopathy and heart failure remain the leading cause of death in Duchenne.…Continue
Added by Kathi Kinnett on September 13, 2012 at 12:00pm — No Comments
Yes, summer is winding down, and the welcoming of fall brings many things – cooler weather, wonderful fall colors, the beginning of school and, unfortunately, the start of flu season. Every year, thousands of people get the flu. While it can be an inconvenience for some, the flu has resulted in thousands of deaths.…Continue
Added by Kathi Kinnett on September 6, 2012 at 9:30am — No Comments
Rick Guidotti has a lot of energy - a lot of experience and expertise, but the first thing you notice about him is, he has a LOT of energy. I first heard him speak at Cincinnati Children's Hosptial Medical Center at pediatric grand rounds. He was in Cincinnati to promote his photography installation (“Positive Exposure”) at the Cincinnati Museum Center. He…Continue
Added by Kathi Kinnett on August 9, 2012 at 9:00am — No Comments
The clear message received by all at the end of the Transforming Duchenne Care meeting, held just prior to the PPMD Connect Conference, was that parents, providers and industry felt a need for clear, consistent standards of care for patients with Duchenne…Continue
Added by Kathi Kinnett on August 8, 2012 at 9:30am — No Comments
"How can parents and patients be assured they are receiving the best care and treatment and what are the components of optimal care?”
PPMD gathered clinical providers, hospital administrators, industry, government, and parents together for a 2-day pre-conference meeting in order to address these critical issues. In the United States and around the world, there is considerable variability in care. Parents often feel it is…Continue
This morning, AVI BioPharma released information discussing their findings thus far in their Phase IIb study of Eteplirsen.
Eteplirsen uses AVI’s phosphorodiamidate morpholino oligomer (PMO)-based chemistry to skip exon 51 of the dystrophin…Continue