A study published this weekend in the journal The Lancet found that an experimental drug called vosoritide increased growth in children with the most common form of dwarfism to nearly the same rate as in children without the condition. The study has raised hope that the drug, if taken over the course of years, can make life easier for those with the condition, known as achondroplasia, including the distant prospect of alleviating major quality-of-life issues such as back pain and breathing difficulties…The study in The Lancet found that children who took the drug grew an additional 0.6 inches on average in one year, with minimal side effects. If taken over many years, vosoritide could produce a substantial increase in adult height, though the study was limited to a year and does not address this possibility, or resolve whether the medication can ease the medical complications common to dwarfism. The trial examined 121 children ages 5 to 17 over a 12-month period. Participants were located in seven countries…“It doesn’t totally restore all of the growth, but it does make a pretty substantial dent in the difference”, said Dr. Eric Rush…Vosoritide uses a synthetic form of a protein that humans produce naturally. It targets the overactive signal that prevents bone growth in children with achondroplasia, said Dr. Ravi Savarirayan.
…But the drug has also ignited a contentious debate in a community that sees “dwarf pride” as a hard-won tenet—where being a little person is an unique trait to be celebrated, not a problem in need of a cure…“I can do everything that someone a foot taller can do, with minor accommodations”, Ms. Schimmel wrote in an email, adding that vosoritide sent a message that those with achondroplasia “are broken.”
…Dr. Savarirayan offered a moving example of what longer limbs could deliver. “We’ve got 12- and 13-year-old girls who now for the first time can do their own feminine hygiene and don’t need to be helped by someone because their arms are longer”, he said…That has produced some milestones that others might take for granted. When her family returned to a water park recently, she cleared the 4-foot height requirement to use a water slide for the first time. “There’s a real confidence that goes with those things”, said her father, Paul Cohen…Yes, Mrs. Mills could get a $900 custom bike so her daughter could ride or teach her to drive a car with pedal extenders, but she will embrace an alternative. “With dwarfism, the world wasn’t built for my child, so if there is something I can do to help her navigate the world a little bit better and on her own, I want to do it”, she said.