A clinical trial published in the New England Journal of Medicine found that children with achondroplasia who took infigratinib once a day for a year grew much faster than those given a placebo.
Achondroplasia is the most common form of restricted bone growth (dwarfism), caused by a change in a gene that regulates how cartilage turns into bone. Children with the condition typically grow at a much slower rate than their peers. The trial tested whether a daily oral tablet — infigratinib — could change that.
After 52 weeks, children taking the drug showed a considerably greater increase in their annualised height velocity — the rate at which they grew in height over the course of a year — compared with children who received a dummy pill. The findings were posted by the New England Journal of Medicine.
The drug is taken by mouth once a day, which sets it apart from some existing treatments that require injections. That said, the research is at trial stage, and infigratinib is not yet a standard NHS treatment for achondroplasia in the UK.
Parents of children with achondroplasia in Kent who want to understand current treatment options should speak to their child’s specialist or GP in the first instance. NHS 111 can also advise on accessing the right service.
Source: @NEJM
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