I’m the worst when it comes to following through on resolutions. Every year on Jan 1st I decide to get in shape, organize my files, and be a better friend. By the 15th it’s clear I’m not getting any of that done. So this summer, when I determined that this would be The Year of Doing Everything, deep down I wasn’t sure I’d have the stick-to-it-ive-ness to pull it off. Making travel plans, researching activities, securing tickets for events, and rearranging schedules is really time consuming and mind-bending, especially when you’re on a budget and your kids go to different schools and your husband’s work schedule is extremely inflexible. It’s a hell of a lot easier to pass up opportunities than it is to take them on! Despite all that, I know it will be even harder if I have to start making sure all of our accommodations are handicap accessible. So I am proud to say that we are really making this happen for Charley (and for our whole family) NOW.
Nothing exemplifies the commitment this takes more than our first outing to a professional sporting event. In September, I decided to take the kids to the men’s finals of the US Open. Thanks to a generous friend, securing the tickets was easy. For this event, thankfully we didn’t have to stress out about the ticket prices or engage in exhausting mental gymnastics trying to outsmart the other eBay bidders. All I had to do was say yes and we had our seats to this coveted event.
That’s where the easy part ended, though. On the day of the finals, Benjy woke up with a horrid flu. I tried to convince him that he was just stressed and tired but the fever and the chills were real, so we had to leave him behind. To make matters worse, the skies were cloudy (dense gray clouds, not white fluffy ones) and the weatherman was predicting thunderstorms. I was ready to give up and watch the match on TV but Charley insisted that the weather in the Berkshires is “totally different” from the weather in New York and it was probably fine in Queens. And even if it was raining we could bring an umbrella. And watching it on TV is not the same. And you promised.
When Charley gets into his unrelenting mode, it is brutal. It’s hard to describe unless you’ve experienced it. Ask any teacher who has had him the classroom or or even his grandparents who (because they’re his grandparents) are inclined to think he is wonderful and adorable and well behaved. They will all confirm this — when Charley gets stuck on something there is literally no way in hell to get him off it. So we got in the car. I’m sure you know where this is going – after the 3 hour drive, we hung out in the rain at the food court for 90 minutes, eating everything in sight. And the match was called and we had to turn around and drive home.
Call me a sucker but I did wake up the next morning and pile the kids in the car to try again. That’s right, three more hours down to New York City because I wasn’t willing to give up. We had planned to see Federer and Nadal duke it out and I wasn’t going to give up that easily. This time the sun was shining, there was no traffic because it was Monday (yup, I let them miss school), and all I could think was This is the day we were supposed to have. Isn’t it wonderful when you don’t let circumstances get you down and you just get right back on the horse. Not even the fact that I had to piggyback Charley up the steep steps to our seats got me down that day. I was too busy congratulating myself for living up to the old adage “If at first you don’t succeed, try try again.”
Toward the end of the first set, when the kids were munching on burgers and I was borrowing my neighbor’s binoculars to scan the crowds for more celebrity sightings, literally out of nowhere the sky became dark. My first thought was “wow it’s already getting dark early” and then I realized it was only 6 pm. I turned my head upward and to my total disbelief a humongous black rain cloud was closing in on us. It came on so fast it was almost unreal. Within minutes the perfect sunny day transformed into a torrential mess. The crowds scrambled for cover and we headed for home.
Believe it or not the kids had a blast, which just goes to prove that any experience can be an adventure. During Take One — after we got soaked huddling under the sunbrella at the food court — a friend invited us to wait it out in a very fancy celebrity-studded suite. I pretended I wasn’t listening while a famous newscaster chit-chatted with an Oscar- winning Best Actress. Charley and Sam stared at NBA star Chris Bosh, then finally got up the gumption to ask him for autographs and a photo op. That experience alone was enough for my boys to justify the 6 hours of car time that first day. During Take Two we got to see some really exciting tennis, even if it was only one set. And of course the kids got to miss school. When they got back home to their friends, those autographs and that photo had my boys floating on air for days.
Knowing it was worth it despite the mishaps has motivated me to keep planning these outings. Next month we are flying all the way across the country for a grand total of two days to see the Clippers play the Lakers and the Celtics, and I couldn’t be more excited for my kids.
Thank you to everyone who has come forward with offers to help us make the most of The Year of Doing Everything. We are loving every minute of it!
Charley is 9 and three quarters years old. When I was a kid looking forward to turning 10, all I could think about was “double digits” and how cool and mature it seemed to have two digits in my age. I felt the same way last year, when my older son Sam turned 10. To have my little boy hit that double digit number seemed like such an important and exciting leap. But as Charley’s birthday looms, it’s not numbers in my head but words — specifically the phrase “wheelchair from 10-12 years old.” I have repeated those words — and continue to repeat them — over and over and over when I tell my story, to teachers, to new acquaintances, at fundraisers. And now my world is colliding with those words. A phrase that became almost rote as I repeated it so many times is all of a sudden fresh and raw and frightening.
We have moved mountains over the past 5 years. If we continue at this pace, Charley and other boys with DMD will surely have not one but several treatment options to extend and improve their lives. Research we got involved in when it was still being worked on in cells and mice has now moved to the clinic. Prosensa’s exon skipping drug is in clinical trials in several European countries. Sildenafil is being tried in 30 DMD boys at Kennedy Krieger Institute in Baltimore. Acceleron and Biomarin are two other companies that have DMD drugs in clinical trials. Remember that when Charley was diagnosed in 2004, there was not one human clinical trial for a promising treatment. The progress is astounding.
I know Charley will have treatment options. But I also know that he is 9 and three quarters, and he has four months to go until he’s 10 and then we’re in that “wheelchair from 10-12 years old” phase. Not every boy with DMD loses ambulation between the ages of 10 and 12. Chronic steroid treatment has prolonged ambulation for approximately two years, giving boys extra time on their feet. And some boys who don’t use steroids stay on their feet longer than average. Others end up using a wheelchair when they are 8 or 9. So I don’t know exactly how long Charley has to continue playing backyard soccer and “balloon ball” (a really dumb game in which he and his sister bop a balloon up in the air and then both of them try to get to it first, nearly colliding every time) and getting in and out of the car without help.
Nonetheless, I’m determined to stockpile some incredible experiences now. Starting today, I have officially declared this the Year of Doing Everything. From Summer 2010 through Summer 2011, Charley and I and the rest of our family are going to do all the things we keep saying we should do, but never get around to. I’m going to focus on Charley’s choices knowing that soon it may be hard or even impossible for him to do the things he’s always wanted to do. We’ll surely be hitting a lot of professional sporting events. Top on his list are a game at the new Yankee Stadium, watching the men’s finals at the US Open (he’ll probably have to settle for 3rd round given our budget!), and any professional basketball game but ideally the Lakers. He has his heart set on a concert featuring either Black Eyed Peas or Jay-Z. And a trip to China or Mexico are both top choices (guess where we’re going?!). This is an APB to all my friends and family and acquaintances — if you have freebies or amazing seats to any of the above that you got through work or through a friend or you can’t use them or you have extras, please think of Charley! A huge priority for me this year is to pack it with fun.
Charley is still bopping around in his usual exuberant manner. He’s tiny for his age (thanks to chronic steroid use) and his ‘fro is way-out (thanks to a stubborn refusal to trim his hair) and he’s been practicing his dance moves, so the overall image he projects is a very cool, compact dude on the move. I’m still betting that all the amazing stuff we do this year will be his firsts of many…and that as a teenager he’ll be climbing the rafters to his seats in Yankee Stadium and standing on two feet grooving at an Eminem concert. I hope we have a year packed with sporting events and concerts and an incredible vacation, and that it ends up being practice for a long life filled with these unforgettable experiences.
Sometimes for a few blissful minutes, I forget that Charley has DMD. To you that might seem impossible, since we’re constantly faced with daily reminders of Charley’s disease. Every evening we follow a rigorous routine of supplements and stretching. Since long walks are challenging, we often have to break out our handicap parking placard. And while Charley’s brother and sister juggle soccer games and karate lessons, we always have to make sure Charley occupies himself with ping pong, board games and other less strenuous “substitute” activities.
Yet despite the significant accommodations we make for Charley on a daily basis, we still sometimes get lulled into feeling like he’s just a typical kid. He has such a passion for sports and outdoor play, and he’s such an energetic little bugger. He is just not the type to sit inside and mope about DMD.
This week, we got a harsh reminder of how fragile Charley is. During a recess soccer game at school, Charley was playing goalie. He almost always plays goalie because running up and down the field is way too exhausting for him. He blocked a kick with his hands and the ball bent his handbackward at the wrist, breaking the bone. After a few minutes of rest and ice, Charley got back in goal to finish the game. But back in the classroom, his teacher could tell Charley wasn’t himself. She called to say, “For a kid who doesn’t generally complain about aches and pains, I can tell something was wrong.”
So to the ER we headed, and sure enough the xray revealed a buckle fracture of the left wrist.
The hardest part is that we just don’t know how to be the best possible parents. We want him to play outside; we want to encourage his “nothing can stop me” spirit…but we don’t want him to break another bone. Charley’s bones are fragile due to chronic steroid treatment, so he is more susceptible to fractures than other kids. At his age, a broken leg could mean the end of ambulation. So it’s pretty scary to give him the leeway to play outside with his friends and wrestle with his brother and participate fully in whatever recess activity he wants to do.
Charley has already started arguing with us about how long he has to lay off the sports. I hate to limit him…I want so badly for his exuberant spirit to prevail. But the broken wrist incident knocked the wind out of my sails, I must admit. With these new therapies so close, I know we have to do everything we can to keep Charley on his feet for as long as possible. The responsibilty of keeping him safe and sound has never felt so weighty.
DMD is heavy duty, and I know the thoughts I express here can be pretty depressing. But you know I will always end on a positive note…We’re lucky that as far as broken bones go, this was a minor fracture. And it was his left arm, thank goodness (he’s a righty)! And two of his 18 cousins also happen to have broken arms this week…so Charley’s in good company. He can’t wait to compare blue casts with his cousin Izzy to see who has more signatures!
I am elated to finally be installed in our new house for so many reasons. First of all, I can now get back to my main job, which is raising money for Charley’s Fund. With almost all of the house-related decisions behind us, a good part of my day is freed up to focus on raising funds. I’ve even become somewhat adept at getting the word out on Facebook, despite my initial aversion to that platform. Speaking of Facebook, Benjy and I are hosting a contest in honor of our upcoming 40th birthdays to see who can collect more birthday donations for Charley’s Fund!
If you want to join the fray but you don’t “do Facebook” (and believe me, I understand that decision), please make a donation via our website in honor of one of our birthdays. On Mar 31 we’ll announce the winner for most donations as well as biggest total collected.
Back to the house…It’s a huge relief to have more space so the kids (and Benjy and me) aren’t in each other’s faces 24-7. Sam, Charley and Maisy are thrilled to have their own rooms. I love and adore my extra deep bathtub. But when you live with DMD, it’s a fact of life that even the most joyous occasions are tinged with sadness. So while we sit in our new living room looking out the huge windows onto the most beautiful meadow and forest beyond, there is a subdued quality to the experience. We do feel lucky to have this incredible place. But no matter how good it feels to be there, there’s always the awareness that we needed this house for Charley because he has DMD and DMD is aggressive and progressive and horrid. While we did a great job of making the house subtly fully accessible, DMD is still very much there with us all the time. A visitor might not notice that the doorways are all extra wide or that the shower in the boys’ bathroom has no threshold or that the light switches are all lower than they would be in a typical house. But for us, those details are all stark reminders that one day our son may need those accommodations because he has DMD and then the reality of DMD is on our minds again.
It is highly encouraging that we are moving so quickly to develop new treatments. With our new program Pilot Trials Now, we are pushing forward FDA approved drugs that are ready to be tried in clinical trials for DMD boys. The first trial will begin this year at Johns Hopkins/Kennedy Krieger institute with sildenafil, a Pfizer drug that was developed for hypertension and angina. Animal testing indicates that sildenafil may significantly help the hearts of boys with DMD, so we are hopeful and excited to get this trial off the ground. Clinical trials are not cheap, so we are grateful to have support for this one from the Nash Avery Foundation, Cure Duchenne, and Hope for Javier — three other foundations run by parents of children with DMD.
Our other research collaborations are moving along too, and we remain confident that a treatment will be developed in time to help Charley. If the next five years are as productive and fruitful as the last five years, we will be in VERY good shape. Of course we need your help to make that happen, so please stick with us as we continue to push forward with every fiber of our being.
This may not sound right but I have to say it anyway — thank god the kids are back in school!!!!! Summer was full and fun but those last two weeks were trying, I must admit. Now that Maisy has started to pipe up and add her two cents into the sibling dynamic, all three of the kids can really get on each other’s nerves!
I shouldn’t let that overshadow the fantastic eight weeks of summer. Charley spent every day at Eisner Day Camp, where he is pleased to be the oldest and — in his opinion — coolest kid in the camp. Charley’s older brother Sam got to spread his wings a little at sleepaway camp in Northern Berkshire county. Although it’s sad to know that Charley won’t join him there, we were relieved to give Sam his much-needed space.
In mid-August, we were lucky to have the best weather of the entire summer for our one-week vacation at a friend’s house in the Hamptons. All we did was lounge by the pool all day long. Ironically Charley was the antsy one, constantly bugging us to play tennis, “volley-balloon,” and card games.
On the foundation front, we spent much of the summer working with Marie Payton, our Clinical Consultant, to map out a plan for implementing pilot trials for FDA-approved drugs. We are excited to have a real roadmap for moving forward. Funds permitting, we aim to have at least one trial start this year and one in the first quarter of 2010. Other collaborations are moving forward on track, including the work at AVI Biopharma and Dr. Jim Ervasti’s research at the University of Minnesota. Because these and other novel therapies are so close, it is essential that we identify FDA-approved drugs that can help DMD kids stay strong until new breakthrough treatments are available.
One strategy for coming up with the funding for these exciting projects was to reach out to donors who have not been active for over a year. So far the response has been encouraging. People who helped us get off to a strong start are now coming through once again to help us push the ball even farther down the field. Thank you to everyone who answered our plea for help this summer!
Please get involved this fall –
come to our Spa Day on Sept 25th (bring a friend on the half-price companion ticket!!)
do your holiday shopping at Live and Let’s Give the weekend of Nov 6-8 in Pittsfield
make a donation in someone’s honor…we’ll send a cute card!
This fall marks many new beginnings: the Jewish new year, Charley’s birthday, we move into our new house, and the initiation of Pilot Trials Now. I have a good feeling about all this…
We’ve definitely entered a new phase in terms of Charley’s awareness of his disease. We are transitioning out of the “honeymoon phase,” when the limitations of DMD haven’t really set in. Until recently, we could kinda sorta blend in with the crowd. Our evening routine has always been tough: In addition to the usual bedtime routines Charley has to take 14 pills with a powdered mixture and then the “yucky drink”, a soy-based food supplement from China that smells and tastes pretty much like sewage. We also have to stretch him for 15 minutes every evening. And in the mornings before school — amidst the mayhem of frozen waffles and unruly bedheads and socks that don’t match — we throw in 12 pills plus a morning dose (2 oz) of the yucky drink. So while these rituals that start and end the day have always been intense, in between we have been lucky enough to function almost like a typical family. Charley is mainstreamed at school, and for the most part he enjoys the same activities as other kids. He has to limit his sodium and sugar intake and he has to rest when he gets tired, but other than that he has been keeping up great.
Over the past few months, however, Charley and his brother have been developing more of an awareness of Charley’s limitations. Just the other day, they were playing with a soccer ball in our backyard. Sam had to retrieve the ball for the 10th time, and in frustration he yelled “I hate this!” Thankfully he quickly clarified his outburst. “I don’t hate YOU, Charley,” he explained. “I love you. I just hate your condition.” It took a while for Sam to understand his feelings of anger, and in fact it is still a “process.” Sometimes his anger is directed at Charley himself, and that is really hard for everyone.
Charley has always been less of an intense personality than his older brother Sam. If he is angry about having DMD, that anger has yet to surface. He does express sadness, and that is very hard for me to bear. This winter he was devastated that he could not ski. One Saturday, I made the idiotic mistake of bringing him with me to the ski shop to buy a new pair of gloves, as we were on our way to go sledding and he had lost his umpteenth glove of the season. We ran into a family we know, and all three kids were booted up, clearly on their way to the local ski mountain. Then a friendly young salesman said, “Hey buddy, are you ready to hit the slopes?” Charley just stared at him, and when we left the store he burst into tears.
We adapt with the times, though, and as soon as the weather improved we bought Charley a bright red electric Razor scooter so he can keep up on the bike trails. Charley’s new ride has brought back the smiles, at least for now. On a recent trip to the Franklin Park Zoo, he had more fun zooming around the crowded walkways than he did looking at the animals. The highlight of this outing for Charley was practically taking out three toddlers as he zipped past them at top speed. The fact that scooters are not allowed in the park – only he was allowed — made it all the more exciting.
I’ve got lots of adventures planned for this summer…elderly ladies and toddlers beware…Charley is on the loose!
by Tracy
Two colorful packets arrived in the mail yesterday, one for Charley and one for his older brother Sam. Sammy’s envelope contained a slick 2-pocket folder stuffed with colorful inserts — promotional meterials from a summer sleepaway camp we’re considering for him. Sam ripped through the pages, shouting “They have windsurfing! They have a zip line! I am definitely going there!!”
Charley’s envelope was also a glossy folder with colorful inserts, but his came from Mass General Hospital; it was an information packet for new patients. The hospital sent it to us in anticipation of our upcoming appointment with a pediatric endocrinologist. Chronic steroid treatment can stunt growth, and Charley hasn’t grown in over a year so are considering treating him with a daily injection of human growth hormone. His growth has stalled so much that he is by far the smallest kid in the class and he shares a shoe size with his four-year-old sister Maisy. Much more alarming as far as Charley is concerned — he can’t go on any of the rides he likes best at amusement parks because he never meets the height requirement.
As the boys grow older, the reality of Charley’s existence is hitting me in the gut more often and with considerably more force. After Charley got into bed last night I sat down with Sam to view the camp’s promotional DVD. Within minutes, he was 100% sold. All it took was a couple of video clips of kids waterskiing, spinning the pottery wheel, roasting marshmallows and screaming “I love camp!”
When the video wound down, Sam looked at me and asked “Mom, why are you crying?” Luckily it was easy to come back with “I’m gonna miss you so much.” That reply was true, but the tears were laden with much more than wistful thoughts about how quickly our kids grow and leave us.
I feel grateful that we can give Sam the opportunity to go to a posh sleepaway camp where he can get away, enjoy the activities he loves, and make new friends. But if I’m being brutally honest, I’m also heartbroken that Charley will not have the chance to enjoy that experience. And I am stressed about finding something he can do that will keep him occupied and happy all summer.
Speaking of occupied — and here I will break into the good news — we are now extremely busy making plans to move forward with our research that has resulted in promising findings. To begin with, Marie Payton, our new Senior Clinical Consultant, is reviewing FDA-approved drugs and some nutritional supplements that show promise as a treatment for DMD. She is working with Dr. George Vella and a pharmacologist consultant to determine which of these treatments are ready for human clinical trials. Marie will outline a clear strategic plan for getting trials up and running. Our goal is to generate trial-based evidence that will help doctors determine which existing drugs should be prescribed for DMD boys to help them maintain strength until new therapies are developed.
That day is not very far off into the future. In fact, several new therapies — such as a drug called Ataluren and a treatment called exon skipping — are already in clinical trials. In addition, several biotech companies and scientists we are funding are on target to begin clinical trials of exciting new therapies within the next 2-5 years. This is not a pipedream. This is not basic science that may one day in the far off future lead to an exciting discovery. Every project we support has a clear scientific plan with a budget and timeline. The question that guides us is always “When will this be ready for clinical trials?” Of course science is somewhat unpredictable, and along the way plans can change. However we work with our researchers to ensure that they keep their eyes on the prize. And of course we all know that the ultimate prize is an effective treatment for all boys with DMD.
If you were not able to donate to our 2007 annual appeal, please consider making a donation now on our “Donate Now” webpage. Our 2007 appeal was down 44%, so we have a lot of ground to make up. Preparing for and implementing clinical trials is a very expensive prospect. Thank you for your support. Please stay tuned for more news of our progress.
When I look back to review previous blogs, I realize how huge the chasm is between what goes on in our lives and what gets conveyed to our friends and supporters. I’m averaging a blog entry every three months, which would seem to indicate that there isn’t much news to report. The truth is that our days are so jam-packed that exhaustion and brain-overload is what prevents me from writing more frequent updates. I consider it a positive sign that we are so busy. We are constantly in touch with scientists who are moving their research forward. We are working with the Darius Goes West crew to raise funds for our fight all over the country. We are collaborating with other foundations to leverage our support to have the biggest possible impact. Over the past four years, the work we do has dramatically intensified. It’s a constant battle to do as much as possible without getting swallowed up whole by this intense fight.
I am very excited to have help in the form of Cheryl Rosen, our new Director of Development and Operations. Cheryl will oversee our fundraising efforts and ensure that our day-to-day operations run more smoothly. With the addition of Cheryl to our team, we are increasing our capacity to support everyone out there who wants to organize a fundraiser or get involved in some way. In this difficult economic environment, so many of our supporters have had to cut back on expenses, including charitable giving. To compensate for the fact that donations are smaller, we need to reach out to more people. That kind of effort is very difficult — if not impossible — with no staff. Cheryl has raised money as a volunteer for various New York- based organizations, including Horace Mann School and the Ittelson Center. Her professional background as a consultant and analyst will inform her efforts as she develops a strategic fundraising plan for Charley’s Fund. Please feel free to contact Cheryl at carosen@charleysfund.org to say hello. She is enjoying getting to know our supporters!
We’re looking forward to a family vacation in Florida next week. During this icy and snowy winter, we’ve been living in a rental house perched at the top of a precarious driveway. Getting down the driveway to meet the bus in the mornings has been an adventure in itself! It’s been a long winter so far, made longer by the fact that Charley is not skiing this year. We are trying to preserve as much muscle mass as possible, and excessive exercise can work against this. While his older brother Sam takes snowboarding lessons, Charley’s been great about finding alternate activities. But it breaks our hearts to hold him back from a family sport we all love.
We can’t wait for some sunshine over President’s Week. We’ll hit the beach and the pool and the burgers. Hopefully I’ll even be refreshed enough to write a post-vacation blog, including some photos.
Charley’s birthday is tomorrow, always a day of extreme mixed emotions for me. No mixed emotions for Charley, thank God…he is 100% thrilled. Charley can’t believe his luck that there is no school on his birthday due to Veteran’s Day. He has already planned his entire day, which will begin with waking up at the crack of dawn to open presents. He then plans to play Mario Brothers Kart on the Wii ALL DAY LONG, stopping only for a lunch break at his favorite eatery, the Great Barrington Bagel Store. Evening will bring cake and more presents, including a first for the Seckler family: Charley’s older brother Sam actually spent his own money to buy a gift that he picked out especially for Charley.
To see Charley in pure heaven does fill my heart. (Thank goodness all it takes is a great bagel and some Wii to make him happy!) But the pronounced passage of time marked by his birthday is frightening. As Charley gets older, we are facing more difficult situations in daily life. Due to chronic steroid use, Charley’s bone density is very low. This year he will not be skiing, because a broken leg is a risk we cannot afford to take. Breaking that news to Charley was not easy, as he is a huge sports-lover and skiing is his favorite activity. The daily steroid treatment has also stunted Charley’s growth, and he is grappling with the emotional effects of being the size of an average five-year-old. It’s tough for him to have the same shoe size as his four-year-old sister, while his older brother is growing and developing normally. In and of themselves, these daily emotional struggles are rough but bearable. It is what they represent that frightens me…the inevitable progression and heartbreak of DMD.
DMD’s aggressive timeline is what keeps us working day and night to implement our well thought-out strategy for developing a therapy for the disease. We are making unprecedented progress against Duchenne, and for the first time in history, the hope for a treatment is very real. We are thoroughly determined to reach our goal and cautiously optimistic that we will get there in time to help Charley and so many other boys we have met over the past four years, as well as thousands more we have not met.
The end of the year is a big fundraising push for us, and this year we have some effective new ways to bring new supporters into the fold. Later this month we will release a short video introducing viewers to our kooky little boy and our fight to save his life. Now instead of telling people about Charley’s Fund in your own words, you’ll be able to send a link to this video, which shows Charley in all his quirkiness and explains how we are making headway against the disease that threatens his life. We’ll send you an email when the video is ready.
In December we are sending out our brand new Gift Catalog. The catalog includes the classic Charley’s Fund merchandise (ie, the original Good Charma Believe Bracelet) along with some fabulous new items. For many of us, asking friends to shop from a fantastic catalog is a lot easier than asking friends to donate money to a cause. It is our hope that you will share the catalog with friends, and encourage them to purchase holiday gifts that support Charley’s Fund.
Finally, our annual appeal and newsletter will be mailed out to all of our supporters later this month. Please do what you can to support us this year. I know it is a difficult financial time for almost all of us. Please keep in mind that we are different from many other charitable causes in that we are on a very intense timeline. We cannot afford to experience a dip in fundraising and then just make it up in years to come. Too many young lives hang in the balance.
Our vacation week in the Hamptons could not have been more glorious. For the first time in a very long time, the weather was our best friend…sunny, clear blue skies with not a hint of humidity. For some reason, beach memories are extremely powerful for me. For those of you who have seen the movie Darius Goes West, you know I get emotional when I reminisce about a trip to Florida several years ago, specifically when I describe Benjy tossing Charley and Sammy around in the ocean. I’m not sure what it is about the sea…maybe it’s a reminder that whoever created DMD also has the power to create such astounding beauty. Or maybe it’s my childhood memories of Atlantic beach, jumping the waves with sisters and friends until dusk, and the dread that Charley’s wave-jumping time is limited. I think the sheer power and expanse of the ocean is humbling and awe-inspiring…makes me think about our place in the world, that we’re all pretty small in the grand scheme of things. At the same time, watching the waves crash in reminds me that our ability to achieve is limitless. We’ve sailed across the ocean, explored the bottom of the ocean, even flown over the ocean…surely we can beat Duchenne muscular dystrophy.
After dropping the kids with their grandparents and the puppy with a friend, Benjy and I headed down to Philadelphia for the most comprehensive annual DMD conference in the world. We participated in two intense days of scientific presentations as well as “break-out sessions” covering day-to-day issues that DMD families have to face, such as physcial therapy and social isolation.
I have been to this DMD conference four times. My first time, in 2005, I remember hearing anxious parents talk about how the scientists seem to say the same thing year after year. That scared me — talking with experienced DMD parents who felt like nothing was changing. Thank God I do not have that feeling now. This year’s conference was markedly different from past meetings. For starters, there was a panel of speakers from the biotechnology industry discussing the different therapies they are developing. Two of the speakers discussed human clinical trials that are underway, and others presented their plans to begin trials in the near future. The excitement over PTC-124, the first DMD drug to enter human clinical trials, was palpable. The company that is developing this medicine just struck a licensing deal with Genzyme, a major biotechnology company in Cambridge, MA. Finally, the biotechnology industry is paying attention to DMD, and the money you have generously donated is being leveraged by big investments from well-known industry players.
In closing, I feel the urge to come full circle with the beach metaphor…something about how the waves have been building, and now they are starting to crash into shore. It’s in my mind somewhere, but as I learn more about this new blogging genre, I realize that it’s better to write more often and less perfectly than to wait three months to produce an elegant essay laced with well-crafted metaphors.
One thing I will continue to do, no matter what, is to end on a high note. So here it is: Charley is still wave-jumping. With a humongous smile on his face, he readied himself for the break, bending his knees in anticipation of the onslaught. Time and time again he cleared the incoming wave with a small but real two-footed jump. And when he got tired, he decided that it is just as fun to swim under the waves as it is to jump over them. It’s amazing that he can still jump, and just as amazing that he can adapt to what life sends his way without a hint of frustration or anger.
With your continued support, we will keep him going until we reach our goal.
I know it will be even harder if I have to start making sure all of our accommodations are handicap accessible. So I am proud to say that we are really making this happen for Charley (and for our whole family) NOW.
