Dwarves at odds over drug that can treat condition

Dwarves are divided over the prospect of a drug that could put an end to the condition by helping children with the genetic abnormality grow at almost the normal rate.

An American Biotech firm, Bio-Marin, has submitted the results from trials of vosoritide to US and European regulators, making its release possible within months.

Dwarfism, scientifically known as achondroplasia, is defined as an adult height of four feet 10 inches. It affects around one in 25,000 infants.

Vosoritide was shown in tests to dramatically increase the growth rate in children with the most common form of dwarfism, according to a study published on Saturday in The Lancet.

Trials were carried out at hospitals at 24 sites in seven countries – Australia, Germany, Japan, Spain, Turkey, the USA, and the UK. Scientists recruited 121 children to participate, with half receiving the drug and half receiving a placebo.

The tests 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, scientists reported, vosoritide could produce a significant increase in adult height – although the study was limited to a year and does not look at other common health problems associated with dwarfism.

Vosoritide utilises a synthetic form of a protein that humans produce naturally.

It targets the overactive signal that prevents bone growth in children with achondroplasia.

The issue of drugs to combat dwarfism is controversial in the United States.

Mark Povinelli, president of the Little People’s Association, told the New York Times that vosoritide “is one of the most divisive things that we’ve come across in our 63-year existence”.

When the group formed in 1957, there were no treatments in the United States to increase height.

The organisation was formed to change how people saw dwarfism, and to help those with the condition live a fulfilling life free of prejudice.

His group now has 8,000 members.

“We want to show that you can have a completely fulfilling life without having to worry about growth velocity,” he said.

 More than 80 per cent of those with achondroplasia are born to parents of average stature, and a child with the condition has a 50 per cent chance of passing it on.

Melissa Mills, of Jacksonville, Florida, who does not have the condition, said she had already decided that her four-year-old daughter, Eden, would use vosoritide if it was approved by the Food and Drug Administration.

“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.

The scientist who led the trial, Dr Ravi Savarirayan of the Murdoch Children’s Research Institute in Melbourne, said the drug could make life-changing differences to those who took it.

“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.

However, Megan Schimmel, who like her husband, Jeremy, and two-year-old daughter, Lily Grace, has dwarfism, said she is uninterested in the drug.

“I can do everything that someone a foot taller can do, with minor accommodations,” she said.

She felt that vosoritide sent a message that those with achondroplasia “are broken”.

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