Sunday, July 15, 2012

Forward progress......

As a board member going in to the 2012 Little People of America Conference in Dallas, I knew one of the most significant issues at the board meetings and at the conference would be that of medical research.  The stage was set at the conference in 2011.  In Anaheim in 2011 we heard for the first time about groups like Biomarin, a pharma company developing what is called a treatment for achondroplasia, and we heard about groups that are raising money that will be directed toward research.  In this case the term treatment means drugs that address certain impairments found in people with achondraplasia, such as sleep apnea, spinal stenosis, fluid in the brain and bone growth.  Little People of America has never been directly connected to research.  However, as the major national organization for people of short stature, LPA would need to be in the foreground on the issue, providing information about updates in medical research to the membership and to the dwarfism community.

In the months just prior to the conference, Little People of America did two things.  First, it created a Frequently Asked Question document that aimed to answer some of the basic questions about medical research related to dwarfism. The document, titled, Emerging research and potential treatments in achodroplasia and other skeletal dysplasias is on the LPA website. Second, just prior to the conference, LPA drafted a position statement.  The statement was approved by the board of directors at the Dallas conference.  It is not yet published on the website.  Basically, in the words of the authors of the statement, it is "strongly neutral."  It stresses the importance of dwarf culture and of the community that is nourished and supported by a group such as Little People of America.  It does not support or endorse medical research but indicates that no matter what choices dwarfs and parents of dwarfs make in the context of medical research, they will continue to find support within Little People of America.

Though I was pleased with the outcome of the two documents, they didn't prevent the membership from getting more involved with the issue at the conference in Dallas.  We have a position statement, but there is still the question of whether or not LPA gets involved in research.  That question comes with a lot of complexity.  The complexity rose to the surface at the tail end of a medical presentation on Sunday morning of the conference.  At the end of the presentation, after members of LPA's Medical Advisory Board opened the floor to questions, a few members from the audience delivered passionate statements about the implications of "treating" dwarfism.  Though treatments are intended to improve quality of life from a medical standpoint, it is hard for some to navigate around the idea that the treatments are changing who we are as a community of people with dwarfism.  With this in mind, the treatments, and discussions around the treatments, can be interpreted as challenges to who we are. 

Also, some people had difficulty with the emphasis on the medical model of the research.  Little People of America is a social support organization, and any venture into the area of medicine should also have a social component.  Yet, there has not been any social research done in tandem with what the community is hearing about Biomarin and medical treatments. This made it very difficult to listen to the medical presentation, because there was little to suggest the social standing of the people who would be "treated" and of the social implications of the treatment on the entire community.

As the conference progressed, it became clear what would be expected of Little People of America.  Again, though LPA is not involved in research, it was agreed that the Medical Advisory Board and that at least one of the groups raising money for medical research would look to LPA for guidance when it came to research.  Though this is new to the organization, it's probably a good thing.  It's better that LPA try to figure out what is going on, then make recommendations on research that would potentially benefit our membership both medically and socially, rather than stay silent and allow things to proceed without our input.  It won't be easy.  This is new territory.  But it's territory about which we should be aware, and perhaps even into which we should venture.


  1. A very interesting read, quite the conundrum indeed. At a glance, it looks like Biomarin are attempting to treat the symptoms (sleep apnea, ear infections, etc.) without removing the cause (the dwarfism itself). And that seems like something the LP community can and should get behind. But when you really read it and think about it, many of these symptoms they're attempting to treat are those related to the restricted bone growth and allowing all the bones to grow at the same, "normal" rate.

    So it raises the interesting question: if someone grows to a height of 5'7" and doesn't have any of the other typical characteristics or traits of dwarfism, but still technically has a dwarfism gene... are they still considered a dwarf? Would they be better off if they'd simply had the dwarfism gene removed/"cured" completely, or is there practically no difference at that point? And of course this would all just be the first step in what could be a series of developments that all lead down a massive moral quandary for the LP community, and really humanity as a whole.

    As someone of average height and no direct relatives with dwarfism (though plenty of friends), I don't know how I should feel or where I should stand on this issue. I'm sure in the coming months/years, many will be looking to LPA for guidance.

  2. Hi Gary. I stumbled upon your blog whilst searching for an answer to a problem I am experiencing with my nine year old daughter with morquio syndrome. I know that this comment section isn't the appropriate place to post my question but was hoping I could contact you via email. My email is My daughter has morquio syndrome and is only 96cm tall at nine years old and is unlikely to get any taller. I'm looking for some advice as to how she can ride amusement park rides. She's not a daredevil, but has been turned away from "kiddie" rides and water slides that have an arbitrary 100cm height restriction. She just wants to go on things that other nine year olds do. We live in Australia but are travelling to the USA at Christmas and would like to take her to Universal Studios. Is there some sort of "work around" the height restrictions at theme parks? Is anyone able to point me in the right direction to find out? The look of disappointment on her face is heart breaking each time she is forced to stand up in front of those "you must be this tall to ride" signs. I apologise for contacting you in this forum and hope that you or your readers may be able to give me some advice.

    Kind Regards

    Danielle Callow

    1. Hi Danielle. Sorry I am not responding until now. I've heard similar stories to your story from others. It is a tough situation. Some people have been successful at earning an exemption from the height requirement from an amusement park or a water park. Typically, they talk to the management and explain the situation. Sometimes it works, but it is not a guarantee. I would advise reaching out to universal studios in advance of your trip. Explain your situation and ask for an exception to the rule. Sorry I don't have better advice.