With deficit reduction remaining a top priority in Congress and a $1.2 trillion spending reduction on the horizon, 2012 will be a critical year to protect Duchenne research and public health priorities. For this to happen, we need your voice on Capitol Hill now more than ever before. Whether that be by joining us in Washington at the Annual Advocacy Conference or advocating from home by contacting…Continue
Added by Ryan Fischer on January 5, 2012 at 2:00pm — No Comments
I wanted to share with you a few of my resolutions for 2012...mainly goals for PPMD and the Duchenne community, but also a couple of personal reflections. 2011 was an incredible year for Duchenne. We made great strides in research, care, advocacy, and in raising awareness. But there is still so much I want to tackle...we all want to tackle.
Read over my resolutions and then please take a moment to share some of your own. This time next year, we can all check back and see…Continue
This year, for the first time, we will have a Run For Our Sons team participating in Disney’s 5K race, which allows runners to push someone in a wheelchair or stroller. Sam’s mom, Kris a first time runner, told us that the whole family will be participating this year.
Added by Will Nolan on January 1, 2012 at 3:30pm — No Comments
Google and social networking enable news – both good and bad – to travel far and wide. Google Alerts have informed us that AVI’s stock has fallen below $1.00 and press releases talked about changes within the company. Amazing that we are all becoming experts on NASDAQ and stock prices!
Families participating in trials and others waiting for next exons and next steps, sit on the sidelines wondering and worrying.
I spoke with Chris Garabedian this morning (so…Continue
Some of the most asked questions we get at PPMD are:
Especially this time of year, our loved ones want to get involved and want to give. We want to make supporting PPMD as easy as possible for you, the person asking your online and offline world, to give and for the donor themselves. So we've come up with a few unique ideas to help you help your friends to make it happen!…
Added by Will Nolan on December 16, 2011 at 9:28am — No Comments
This time of year everyone is in holiday mode, or getting into holiday mode, or sick of being in holiday mode. But something else happens this time of year that effects many of us – especially our children. Report cards. The end of the year is when teachers meet with parents to tell them how little Timmy is doing in class. If Timmy is in high school or college, grades…Continue
Added by Pat Furlong on December 13, 2011 at 2:30pm — No Comments
For the holidays, Pat and Ivy have co-blogged about words that are particularly used this time of year. These words are often redefined by our community because our everyday life requires new definitions. But that does not mean that we must be denied any of the joy of the holiday season.
Happiness and Joy
Endocrine issues are deeply personal to every one of us. They represent our masculinity, our femininity. They impact our weight, our height and how we look. As our boys with Duchenne grow and develop into adolescents, they and their parents are faced with these intensely personal and complex issues. The purpose of this meeting was to engage with experts to develop information related to these issues that parents and their sons might use with their physicians to encourage (or insist) upon a…Continue
Redefining traditions. This year, Tom, my girls, my son-in-law, and I chose to gather in New York City for Thanksgiving. Well, "chose" may be the wrong word. New York City is just logistically easiest. The truth is, we would meet up with our girls in Antarctica if it meant we could be together during the…Continue
Like every other profession in the world, there are inside jokes in medicine. Random friends and acquaintances send photos or comic strips characterizing nurses as battle-axes waving syringes or doctors ignoring the patient, calculating their golf game. In other cartoons, women are caricatured over time by the degree their breasts sag.
Today, I received this from a friend:…
Added by Pat Furlong on November 21, 2011 at 10:24am — No Comments
The death of Brady Sherman (14 years old) is heartbreaking and strikes fear in the hearts and minds of every parent of every boy with Duchenne. It is so frightening that the idea of wrapping your son in bubble wrap to protect him or never letting him out of your sight, makes perfect sense.
Today we do not have any concrete answers about Brady. We have some ideas about what happened and have been connecting with experts (pulmonary, cardiology,…Continue
Co-Blog – The following is a co-blog put together by myself and Ivy Scherbarth, a mother from CO and PPMD’s FACES Coordinator for CO/WY. This is a new project we have started where we will both write on similar topics from two different perspectives, but living in the same world of Duchenne.
To Have and Have Not with Duchenne
At the recent World Muscle Society meeting, which took place in Portugal, a great deal of new data on exon skipping was presented, both on the status of current human clinical trials and on the efforts of investigators to improve the efficiency of the technique by combining exon-skipping with gene and stem cell delivery. Below are a few highlights from the meeting with the caveat that it was impossible to cover every poster and talk.
Muscle Stem Cells
Added by Ann Martin on November 3, 2011 at 8:30am — No Comments
HerSelf First. The name may strike you as a bit quirky. Some of you may wonder what exactly does it mean? And others may sigh and think ‘wishful thinking’. We are all technically savvy, able to put mobile phones on ‘silent’ or ‘vibrate’, computers on ‘sleep’ or ‘hibernate’ and have the advantage of using ‘restart’ or ‘reboot.’ But…Continue
The Duchenne community has been eagerly awaiting news about a Phase 2 Randomized, Placebo-Controlled, Multiple-Ascending-Dose Study of ACE-031 in ambulant, steroid-treated boys with Duchenne (A031-03). We were all disappointed to hear the study was terminated and hoping to understand what were the results of the study in terms of safety and pharmacologic effects and what expectations we might have with regard to next steps for ACE-031. We needed a therapeutic dose of…Continue
We have a very busy weekend ahead of us, with some fantastic events happening in the community!
I wanted to take a moment to highlight these fun and very different fundraisers taking place over the weekend.
Big shout out to all those who are organizing events (not just these events- all…Continue
Added by Ryan Fischer on October 21, 2011 at 1:30pm — No Comments
The MD-CARE Act was signed into law in 2001 and reauthorized in 2008. What’s that got to do with a clinic survey you might ask? The MD-CARE Act was a pivotal moment. In a certain way, it was…Continue
Co-Blog- This month, I wanted to share with you a blog Ivy wrote that really affected me and I think will move you. For me, this is story is about taking off our glasses, the way we view others, the way we view the world. Hard as it is, heartbreaking as it may be, we have to squint, to see the world differently, and to see the world as our sons see the world. And with some luck, we might just appreciate ‘the blue one.’ Thank you Ivy for sharing a moment we all can…Continue
As we say goodbye to September and hello to autumn, I wanted to take a moment and thank you.
PPMD and the entire Duchenne community are busier than ever. There is so much to do, to push forward, to get done, in our daily lives and in the care of our sons. Every now and then, though, it is important to take a minute to exhale and realize what you’ve done, what’s been accomplished, and what is happening around you.
Added by Pat Furlong on October 5, 2011 at 9:00pm — No Comments