Drug to boost growth in kids with dwarfism shows promise
A drug that helps regulate bone development has been found to boost
growth rates in children with achondroplasia, the most common type of
dwarfism, in a global trial.
The patients’ average boost in height to about 6 cm (2.4 inches) per year was close to growth rates among children of average stature, and the side effects of the drug were mostly mild, said study co-author.
Right now, the results of the study show an impact on growth, and this effect is sustained, at least over nearly four years in this trial,” he said.
Results of the phase-2 trial showed that the drug, vosoritide, was generally well tolerated by patients.
On average, participants in the trial grew at a 50 per cent faster compared to baseline with no adverse effects on body proportion, the results showed.
Achondroplasia is caused by over-activity of a signal that stops growth, and could be likened to overwatering a plant, said lead author.
Achondroplasia is a genetic bone disorder affecting about one in every 25,000 infants.
It is caused by a mutation in the FGFR3 gene that impairs the growth of bones in the limbs, the spine, and base of the skull.
The most common health complications experienced by children with achondroplasia are spinal cord compression, spinal curvature and bowed legs. About half of these children will need spinal or other surgery.
Unlike other treatments – such as growth hormone and limb-lengthening surgery – that focus on symptoms, vosoritide focuses on the underlying cause of achondroplasia and directly counteracts the effect of the mutation that slows growth
The patients’ average boost in height to about 6 cm (2.4 inches) per year was close to growth rates among children of average stature, and the side effects of the drug were mostly mild, said study co-author.
Right now, the results of the study show an impact on growth, and this effect is sustained, at least over nearly four years in this trial,” he said.
Results of the phase-2 trial showed that the drug, vosoritide, was generally well tolerated by patients.
On average, participants in the trial grew at a 50 per cent faster compared to baseline with no adverse effects on body proportion, the results showed.
Achondroplasia is caused by over-activity of a signal that stops growth, and could be likened to overwatering a plant, said lead author.
Achondroplasia is a genetic bone disorder affecting about one in every 25,000 infants.
It is caused by a mutation in the FGFR3 gene that impairs the growth of bones in the limbs, the spine, and base of the skull.
The most common health complications experienced by children with achondroplasia are spinal cord compression, spinal curvature and bowed legs. About half of these children will need spinal or other surgery.
Unlike other treatments – such as growth hormone and limb-lengthening surgery – that focus on symptoms, vosoritide focuses on the underlying cause of achondroplasia and directly counteracts the effect of the mutation that slows growth
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Labels: anchondroplasia, bone disorder, boost, Children, dwarfism, genetic, growth
posted by G S Iyer at 3:21 AM
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