Hope for children with achondroplasia, “the most frequent form of dwarfism”, report The Journal of Montreal. A drug to improve the growth of people with dwarfism has been approved by US authorities. It will “have to meet an unmet medical need for more than 10,000 children in the United States,” explained Theresa Kehoe, head of the US Medicines Agency (FDA).
1.57 centimeters more, this is what this treatment marketed by the BioMarin laboratory would allow. According to its clinical trial conducted on 121 people aged 5 to 14 years, participants who received Voxzogo grew 1.57 centimeters taller compared to those given the placebo, says the FDA. This drug should be administered as a subcutaneous injection once a day, it was specified in a press release published on Friday, November 19.
Treatment already authorized in the EU
As a reminder, a child over 15,000 to 40,000 births worldwide suffer from this genetic disease. The height of an adult with achondroplasia averages about 1.2 meters.
“More than a decade of scientific research supports the medical breakthrough represented by Voxzogo“, rejoiced Jean-Jacques Bienaimé, CEO of BioMarin. Note that these tests revealed side effectsincluding vomiting and hypotension, which is a drop in blood pressure which may cause dizziness and fainting.
This treatment should be available in the United States from December whenit has already been authorized in the European Union since this summer. Voxzogo is also “under review in Japan, Brazil and Australia,” says an article in the daily West France.
A scientific discovery that is not unanimous since some fear that this type of care accentuates the stigmatization towards people with dwarfism, too often considered “abnormal”.
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