"Experimental drug to treat dwarfism raises questions" by Michelle Fay Cortez and Caroline Chen Bloomberg News July 04, 2015
An experimental drug to treat dwarfism by lengthening children’s bones is meeting skepticism from the leading group in America that represents small-stature people, which questions giving such a medication to children.
That makes you some sort of conspiracy kook, right?
Parents like Chelley Martinka, whose daughter has the condition, achondroplasia, say they have no intention of giving their children injections of BioMarin Pharmaceutical’s BMN 111. That won’t change even if the drug lives up to the promise of its early trials, said Martinka, who blogs about her daughter’s condition at ‘‘A Is for Adelaide.’’
‘‘My daughter is incredibly smart, she’s funny, she is the most loving person I’ve ever met,’’ said Martinka, who doesn’t have achondroplasia. ‘‘I can’t ask for anything else. To do something superficial, to give her a couple of inches, it’s ridiculous.’’
BioMarin is betting it can persuade parents to embrace the drug’s potential to add height and avert health issues that come with the genetic disorder. The medicine will be a hot topic at the annual meeting of Little People of America, which started Friday and runs through July 10 in St. Louis.
Sorry I'm so sour, but that is what is wrong with AmeriKan medicine these days.
Leaders in the organization are talking regularly with San Rafael, Calif.-based BioMarin. They worry that the drug is being presented as a way to address an ‘‘affliction.’’
‘‘Just because a person has achondroplasia doesn’t mean they are going to have health issues,’’ said Gary Arnold, the group’s president, who encourages new parents to get to know others in the community. ‘‘This drug might be right for some people, but we also think it’s important to get across the message that it’s not necessary to live a healthy and productive life.’’
Just don't make any rash decisions, okay?
The average man with achondroplasia grows to a height of 4 feet 4 inches while the average woman with the condition reaches 4 feet 1 inch.
I'm not trying to be flip, really I'm not, but without them there are no Munchkins.
If the drug is effective for children across that age range, that would create a market of as much as $1.8 billion a year, estimated Robyn Karnauskas, an analyst at Deutsche Bank, in a presentation to clients.
What's worse, my truly loving insult if taken as such (sorry), or being exploited by these guys who are the ones pushing inadequacy and freakishness upon you -- so they can make a buck.
We are all the way the Good God made us, and ALL LIVES MATTER!
Michael Yee, an analyst at RBC Capital Markets, estimates that BioMarin could charge $300,000 for the drug, which would be the company’s biggest product....
Don't worry; taxpayers and consumers, 'er, patients will fork all that over to Big Pharma.
There was no placebo comparison, so the trial didn’t examine how they fared compared with children getting no treatment.
There is no evidence so far that the therapy made the proportions of the children’s bodies more like an average person’s. And there is no way to know if the accelerated growth would stop, a potential problem since other drugs tested to treat the condition have been difficult to control.
Looks dangerous, but what the hell?
Maybe they are transheight.
Look, the pharmaceuticals are finally preying on the little people and their insecurities! Looks like they've finally been accepted!
"In their quest to find ways to give their daughters extra strength and more time, the Kennedys are willing to accept far more risk than if they were simply trying to treat an ordinary disease of childhood. While these types of patient experiences are powerful, medical professionals worry that such arguments will be used to erode a carefully wrought standard of evidence that today helps to avoid the tragedies of the past, such as the birth defects caused by thalidomide in the 1960s and infertility caused by the Dalkon intrauterine device in the 1970s."
It's all bu$ine$$ now.