Disability Pride Parade, a video.

Caught this video on Saturday in Chicago at the Disability Pride Parade, http://www.youtube.com/watch?v=-EBR_CJz6EU.

Just in time for the birthday of the Americans with Disabilities Act.

// Catherine Calhoun

Parents helping kids and kids helping parents, a meet in the middle.

I just returned from a meeting in Europe and one of the best things about these meetings is hearing stories of families, listening to experiences, and learning about those special times when a child’s insight makes us stop in our tracks. It is not about criticism or what is wrong, right, or in between. It is just about those times when we find our hearts’ making a recording that we will remember all the days of our life.

One mom talked about all the things she was doing for her son. She described a pretty time consuming schedule that included supplements, stretching exercises, aqua therapy, as well as a very strict diet. It was pretty amazing really and I had the sense that she had achieved the perfect “10” as a mom, someone we would all look up to and wonder just how she fits everything into a 24-hour period or if she found some magic to stretch the few hours in a day. All of a sudden in the middle of her story, she had tears in her eyes as she talked about one particular evening when her son was grumpy as she started the stretching exercises. He had had a full day. As she started his stretches, he started crying. She asked if the stretch was painful. He said ‘no.’ Like all of us, she probed, asking if something went wrong at school, if his stomach was upset, if he was in pain, looking for something, anything to understand the tears. We have a natural tendency to try to ‘fix’ everything. He simply said he did not want to do the stretching tonight. With that, her fear increased and she tried to sooth him, tell him how important this was for his muscles, how much she was doing to help him. And in that very simple way that children have, he said, “You are not doing this for me, I am doing this for you.” She felt her heart breaking.

I also spoke with a father whose son is now in college. He discussed his son’s detailed agenda to include a variety of interventions squeezed in between college classes and studies. I asked about friends, about movies, about music, about laughter. This father said all that would come later. I wondered when ‘later’ was planned. This father was so worried that if something interrupted their rigid schedule, his son would lose function. He felt this regimen was essential to maintain the life his son had, the things his son was able to do at this very moment. One glitch, one change, and it would fall apart.

I remember hanging on for dear life, thinking that if I changed one thing, if I took time to breathe, time off, time to enjoy something or some activity from my previous life, things would fall apart. It felt like my life was hanging on a thread. I remember hanging everything on the future. I started sentences with ‘when this happens,’ thinking that a certain clinical trial would yield amazing results and then we would catch up on whatever it was that we missed.

Every once in a while, I think it is good to take a step back and remember, the life we are living is not a dress rehearsal, it is the real deal. There are no ‘do overs’ and no way to recover time lost. So, while we are all aggressively pursuing what we think is the best route for our sons and daughters, it is important to listen to their voice, to ensure the discipline of care includes their perspective and their opinions.

And as you make up the schedule, please include 30 minutes of laughter. It should be considered a therapeutic intervention!

Reposted with permission from Pat Furlong’s blog.

{Photo by Bradley Gee, X Marks the Spot}

Learning to balance privacy and the call of advocacy

As with anything, it turns out there is learning curve to using my personal blog as a platform for advocacy. It is a challenge that I am having to deal with the more lately, as the date for my son’s next surgery looms near.

This will be the first major hospitalization since I started chronicling his medical journey for the internets.  To me it’s a biggie, a jaw distraction.

My dilemma:

Do I blog our ENTIRE experience?

Do I upload potentially graphic pictures?

Do I owe my son his privacy or do I owe my readers a REAL glimpse into our lives?

I still haven’t decided.

As a mom, I struggle with questions How much information is too much information? and  Am I violating my son’s privacy?

But as a writer, I am constantly reminded that I don’t do it for myself, I write to help other families. When I think about how much sharing our story, be it through words or pictures, can help other parents and children I feel I am doing the right thing, for us.

Obviously it may not be the right thing for other families. But there is a strong urge to continue living our lives as transparently as possible. Only then will others truly realize what we go through — the good and the bad.

When all is said and done, I just need to find the fine line between his personal privacy and the call I hear to advocate for him and with him. Can I have one without the other? I think so. Surely I can find a way to advocate without plastering his surgical recovery pictures all over the internets.

How do you maintain your privacy when places like Facebook and Twitter are made to keep interested parties updated on your every move?

Photo: Aaron Kohr

Sometimes dogs are the best medicine, for bone pain.

2:32 a.m. That’s the time of night (or super early morning) that Billy wakes up. I hope it’s a bad dream from too much Scooby-Doo. I listen to the monitor for a minute or two to see if he’ll fall back to sleep. I count on my fingers – January, February, March. Not long enough. I was really hoping we’d get to June before he needs more pain medicine for his bones (by IV infusion, about 5 hours).

I plod down the stairs to his room and offer him some water. He quiets. I trip back up the stairs and try to sleep, heart pounding from steps in the dark and worry. He cries out again (repeat above).

He cries out again. This time I just stay in his room pushing the dog into a tighter curl-up at the end of the bed as I perch on the edge of the twin mattress. I can’t really sleep at this point. And think how much I hate bone disease. I hate the surgery scars (make me wince, on the inside, every time I see them). But what I hate the most is the pain.

I won’t know for sure for a few more nights. Fingers crossed it’s just a bad dream.

I wrote this in May but was too superstitious to post it. It was pain, and Billy did more infusions. I was talking with one of my favorite researchers today about the pain. Apparently, Billy has it worse than most. Maybe he feels okay about 80 percent of the time and then miserable with pain 20 percent of the time? That 20 percent haunts me.

One of the reasons we (me) went for getting  a companion dog for Billy was because I read that pets can help with pain, pets make people feel better. Everyone, pretty much, thought I was out of my mind to add another responsibility to my day to day life. Now everyone loves Picasso and has no doubt he is great for Billy, especially when Billy sings songs to Picasso like “you are my buddy, you are my pal” as he gives him sweet pets.

The Council of Mothers (and a few devoted dads)

The Council of Mothers (and a few devoted dads) convenes as necessary in the wee hours of the morning, the midday afternoon on holidays, the Tuesdays after checkups and at all other times deemed necessary by its members. Together we share one another’s questions, worries, and our greatest common fear – the loss of our children. 

Some of its wisest members are the mothers and fathers who’ve already crossed that chasm once and who, instead of turning their backs and continuing their lives in the opposite direction, fix their eyes on those of us still walking this treacherous road and on our children; pouring their hearts, their souls, their experience and all the love they had for their own children into ours.  

Such wisdom only comes from life’s hardest lessons learned, and theirs – regrettably – are lessons they can now apply only to the children of others.  It is their hindsight, their regrets, their “If I had it to do overs” that serve as the only road map available for this journey of ours.

Many in our group serve as surrogates, knowing they’ll have no children of their own because they’ve chosen not to risk passing on the disease that brings us all together. With all their strength and determination, they walk along with us, loving our children as their own – loving us as though we are family. 

There are also several others who, like me, do not carry this disease but who loved someone who did; someone whose grave we stood beside and to whom we said “goodbye” far too soon.  And now, like living in a land where we do not speak the language, we are raising our children as best we can, hoping to choose wisely, and wishing that we bore the pain in the place of our sons and daughters.  

The collective wisdom and experience this Council brings to bear serves as our reference when any of us, as I did recently, face a new experience with our children and have nowhere else to turn.  As is the case with virtually all rare diseases, there are precious few in the field of medicine who, though they may have the best intentions and sincerity of effort, can offer any actual valid guidance. 

At even the most prestigious well-respected hospitals and institutions, very few doctors or surgeons will have seen a child or adult with a body like theirs.  While some may have a vague memory from medical school about Ehlers-Danlos Syndrome, almost none will have diagnosed it themselves — not through any lack in their qualifications as a doctor, but simply as a result of the rarity of the disease.

Sounding the Alarm 

On a Tuesday night a few short weeks ago around 10:00 p.m. I was the one sounding the alarm, posting on our internet support group a message about my son.  For our children, and for the adults living with Vascular EDS, virtually all sports and activities are off-limits.  Because of the high-risk of injury, aneurysm, organ and arterial rupture that follows them their entire lives, very few athletic options are available.   But, in hopes of finding a way around that difficult reality, I’d signed him up for a community swim team that a few of his life-long buddies are on.  He’s always loved swimming and being able to do something he loved in a team environment was definitely a chance worth taking. 

After the first night’s practice he was understandably tired, but he was also excited and quick to tell me how much fun he’d had.  The coaches had already moved him up to the intermediate group where most of his friends were placed and I could hardly wait to share the good news with my friends online. 

But before I’d had a chance to share my excitement, the moment was gone. After the second night of practice, reality crept back in and like the stupor following a night of drunkenness, I felt sickened by the dizzying truth of what his body can actually handle. My son was in pain; his jaw was hurting and difficult to open.  His shoulder, which has become increasingly loose-jointed over the last year, was in more pain than he’d had so far.  He came to me quietly when no one was around and this normally very active boy, who just one night before was bursting with joy and anticipation at the possibility of a team sport he could enjoy with his friends, told me with tear-filled eyes he didn’t want to go back – he couldn’t go back – because the pain was too much. 

By the time he finished telling me about it, I learned it was more than just his jaw and shoulder; it was his knees, his ankles – all of which are dependent on the one ingredient his body cannot give him — collagen.  Aside from his vascular system, his joints will pay the highest price for this deficiency; possibly bringing on arthritis as early as adolescence – as early as now. 

Once the kids were in bed, I grabbed my laptop and retreated to my bedroom – ignoring all other responsibilities for the night.  I sat alone on my bed and went looking for what has now become my lifeline; a close-knit group of people formed over time with others just like me – desperate for guidance and understanding. 

I typed at a furious pace; ignoring the tears of anger running down my cheeks. “How dare this damn disease take something else from him!” was running across the screen of my mind like one of those annoying crawls on T.V.  How was I supposed to handle this?  Should I take him to the doctor?  Did he need to go to the dentist?  Or, was this just “normal” muscle pain from a new activity?  Would taking him to a doctor or dentist even do any good?  What were the odds they would even be able to give me good advice?  What experience could they possibly draw from to help me sort this out?  The stream of words became a flood of anger, confusion, and pure, raw emotion – and without worrying how any of it may have “sounded”, my rational mind was no match for my mother’s heart that was breaking – again – for the losses my son has endured, and will endure, throughout his life.   

Having thoroughly emptied myself into this cry for help, I sat back on my pillows and took a long, deep breath.  Still utterly confused, and now emotionally drained, I could relax just a little knowing I’d sent up my flare; knowing someone would see it, and knowing help would soon come.  

Within the hour I received my first response from a mother in Canada who’d lost her son seven years ago at the age of 13.  He had experienced chronic jaw pain off and on in the weeks before he died from a thoracic aortic aneurysm.  As has been the case in many such messages, regret was the tone of her voice; telling me now what she had intended to do but never got the chance.  She had been concerned about his jaw; she’d planned to take him to the doctor, but then one night it was over.  At home in his bedroom with her son in her arms, she held him as he closed his eyes and slipped away from her.  Her pain was as evident now as it surely must have been that night when the unthinkable settled in on her and her family in the most merciless way. 

She was quick to remind me it didn’t mean that’s what my son was dealing with, and didn’t want to frighten me; but she also felt compelled to let me know the possibility was out there and until I’d gotten her email, I’d never known that jaw pain might be a symptom of something serious.

By the next afternoon and throughout the week, I had more replies than I could keep up with.  Mothers and fathers, husbands and wives, and people who are living with VEDS offered their words of encouragement, caution, and something to lean on while I struggled to find my way through this.

In spite of the distances that separate us, it was as though this Council had convened on a moment’s notice someone’s living room or around a dining room table to figure out this problem as a team.  As the week wore on, my anxieties began to ease with each new message I received.  I read each word gratefully– keenly aware of the price at which their experience had come. Bought with the pain in their own lives and the lives of those they love, paid for with grief and regret, theirs are the words I cannot ignore.  Theirs are the words I must not ignore.

With all the frustrations and disappointments that go along with raising a child with a rare disease, having this group has meant the difference in my level of sanity more times than I can remember.  Whether or not I ever lay eyes on them or find out what their voices sound like, I know without question they are with me.  In the doctor’s office, the emergency room, or in my own home facing another night racked with insomnia–at any hour of the day or night–I can ask for help and someone will be there.   I know I am no longer alone – and next to a cure . . . that’s the most imporant thing.

Great Opportunity!!

 

EDS Today is competing in the Chase Community Giving Challenge on FB to win up to $250,000 for research. 

We WERE in first place for the first five days, but have been bumped down to 5th – and could use your help moving back up. 

The first place charity at the end of the voting gets $250k; the groups in 2nd, 3rd, 4th, and 5th will each get $100k. Goups ranked below 5th place will get $25k. 

You can help us out and cast your vote by clicking on the picture.  You’ll get 20 votes to use in the competition, so look for others you’d like to help out.  After you’ve used the first five votes, you’ll get a “gift vote” and can use it to vote a 2nd time for EDS Today. 

Please share the link and information with as many people as you can.  The competition lasts until July 12th – winners will be announced on July 13th.  We need as much help as we can get generating some buzz and getting those votes coming in! 

Many thanks!

Debbie Vaughan

FDA Database Aims to Spark Orphan-Disease Drug Development

By Amy Dockser Marcus

For months now, the Food and Drug Administration has been trying novel ways of encouraging drug makers to develop drugs for rare diseases.
Today the agency’s Office of Orphan Products Development is launching a new gambit—a database of FDA-approved compounds and products that show promise in rare diseases too.

The concept is called “repurposing” — finding new ways to use products that have already been okayed by the FDA for some other use.

The already-approved products in the Rare Disease Repurposing Database are unique in that they’ve also previously received orphan-drug designation, meaning they’ve shown potential to treat one or more of the diseases affecting 200,000 or fewer Americans. (While orphan-drug status doesn’t guarantee FDA approval as a treatment for the rare disease, it’s a prerequisite for getting incentives such as tax breaks and marketing exclusivity if the agency does okay the drug for that purpose.)

Timothy Coté, director of the orphan products development office, says that testing an already-approved drug as a treatment for a rare disease has significant advantages — for one, it’s already been found safe by the FDA. And running trials on an existing drug is much cheaper than trying to develop a totally new compound.

Despite the incentives in the Orphan Drug Act, there are only about 350 such drugs that have been approved — and there are some 7,000 rare diseases.

Information in the database is already publicly available through the FDA but until now has been scattered. Coté says officials went through more than 2,000 orphan drug designations to compile the new 235-drug database, which he says is likely to be used not only by drug companies but also by venture capitalists looking for promising investments and patient advocacy groups eager to drive drug development for their rare condition.

Coté says he hopes the new database will spark drug development efforts. But he plans to do some repurposing of his own. He says the next step after the database is released is for his office to carefully study the list, identify some particularly promising products, and urge the companies to move forward.

Link to article

Father’s Day Turns 100!

We know some amazing dads who work so hard caring for kids with rare diseases. This year we want to celebrate them!

Father’s Day turns 100 on June 20, 2010.

EXCLUSIVE: Pfizer Plans to Move Fast on Rare Disease Pacts

Pfizer went public with its new rare disease unit earlier this week, but one of its top execs has been working away for the past few months setting up a division that is preparing to team up with a range of biotechs on new therapeutics.

“This morning at 7 a.m. I was at breakfast, talking to a company,” Jose Carlos Gutierrez-Ramos, Pfizer’s Cambridge-based senior vice president for biotherapeutics research and development, tells FierceBiotech. “We are actively talking to people and have been for the last two months.”

Pfizer isn’t just interested in partnering with biotechs working on new drugs for rare diseases. The company also wants to forge close ties with leading academics as well as key patient associations, several of which have taken an ambitious lead in funding studies of experimental therapies. In some cases, three-way pacts partnering Pfizer with a patient association and a drug developer could make sense.

“We’ll do internally what we do well,” he adds, “and what we don’t do well we’ll do externally with others.” Dr. Edward Masciolo, who had founded Dapis Capital, will head the unit and report to Gutierrez-Ramos.

The goal is to create a small, “highly virtualized, highly focused” group that will start inking pacts in the next two to three months as it builds a significant pipeline of new drugs over the next two to three years, says the Pfizer exec.

“We are very interested in certain areas,” he adds, highlighting hemophilia, muscular dystrophy and protein misfolding. “There are certain areas where we have biases,” with the seriousness of the disease and the impact Pfizer can make on it the two leading indicators of where it will focus its resources.

But Pfizer isn’t starting completely from scratch, either. The pharma giant has spent years developing new hemophilia therapies and recently licensed Protalix’s Gaucher’s drug, which will put it in direct competition with Genzyme, a large cap company that has made a big mark selling some very expensive medications for rare diseases. Genzyme’s successes with drugs like Cerezyme helped inspired GlaxoSmithKline to start their own rare disease unit back in February.

“We have significant expertise in rare diseases,” Gutierrez-Ramos says. “We’ve worked in hemophilia for 10 years and as a consequence of that we have developed a protein therapeutic and protein replacement capability and know-how that is unlike few other companies.” – John Carroll

Link to article

Chase Community Giving Summer 2010 on Facebook, rare disease ticket.

Community Building Tools, a technology conference recap.

Before I forget everything I learned in early June at a technology conference, here goes.

Use good content to build your audience (and encourage your members to share/make it easy for them to share) – you have to get out the “why” for your organization. Does your content touch people emotionally? Move people to action? Make them different? You gotta have stories and thoughtful analysis.

Sexy concepts like “efficiency and transparency powered by social media” (e.g. Facebook, twitter, etc.) do not replace the old school tools (phone banks, petitions, door knocking, lobby days and email campaigns). The point of online tools is to better connect people with what they need offline – online is not the end, online tools simply help you better deliver on your mission. Face-to-face.

And it’s not about the size of the list (not not not). It’s about – how effective are your supporters? 2,000 believers are better than 100,000 people (followers/fans/likes) who don’t really care.

What about mobile tools? Will “text” raise millions of dollars for your organization and magically build your group? Probably not.

Consider your audience when you build a mobile campaign – do your members rely more on mobile devices like cell phones with very limited internet access otherwise? Regular cell phones and smart phones are not the same – don’t treat them the same for a campaign. Make it easy to opt out (a simple “stop” to remove from the list).

If mobile suits your group, it can be very important to you for (1) list-building (a face-to-face ask for mobile contact information works best – that offline thing again), (2) communication and (3) reaching super volunteers. People at the conference spoke very highly of Mobile Commons. Mobile works for collecting questions (i.e. What would you ask Pres. Obama?) – but you’ve got to follow-up and show participants what you did with the questions, your follow-thru and report back, very important.

Mobile can be powerful. During a rally in D.C. with about 200,000 attendees – the hosting organization put up a big board for posting text messages from people too far away to attend – a chance to text in public support for the rally. 30,000 texts were sent in, how cool is that? Another neat idea is telephone town halls with zip code locate to help bring people together for small face-to-face house parties/meetups.

Don’t expect to gather much for demographics with a mobile campaign. You could make an offline offer (e.g. sticker) that could be useful for gathering demographic details (i.e. mailing address). (Now I know why I get those offers – and I just thought people were being nice).

When you’re building your group you have to be patient.

Sometimes paid ads are needed – you want to be one of the first three finds for a Google search related to you/your mission which means Google AdWords can be very important (especially to a nonprofit). AdWords gone rogue (see Kit Kat plus Greenpeace).

Listen. And treat people how you would like to be treated. Still, expect trouble makers (i.e. online trolls). Don’t expect to control the message top to bottom – social media tools sometimes mean you will lose control of the message as people engage and pass along. You want a balance between participation and police state.

Honesty. You don’t have to know everything.

Measure everything and keep doing the things that work.

Rule of thumb on everything you post/share/send out – keep it 80 percent not about you – I’m talking links, photos, videos and the like that are interesting and worth sharing, something that makes an emotional connection somehow because it’s sad, funny, silly, informative, etc. – that leaves you 20 percent about you (e.g. staff stories and photos).

Don’t drag campaigns out – people get fatigued with campaigns lasting longer than two weeks. Headlines and deadlines are key – if you can find combined relevance in news and your mission/supporters – use it.

Blogs. Nice checklist.

Now you know everything I know (it seemed like more when it was just in my head). For more on technology tools, see Personal Democracy Forum.

// CC

P.S. I love Google for making it possible for me to attend the conference (and giving me a cool yellow/orange hat which I had to give Billy as his New York City souvenir because I was so busy conferencing that I didn’t do any shopping). Next up . . . BlogHer in NYC this August (shopping mandatory).

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