An innovative drug trial has reaped promising outcomes for the children with achondroplasia which is a highly common form of dwarfism, make it likely for them to grow at almost the same rate as the normal children’s height.
Clinical trials were led by Murdoch Children’s Research Institute and its results were published on Tuesday in the New England Journal of Medicine.
The other component of the research, which is created, as well as, funded by the United States based biotech company ‘BioMarin Pharmaceutical’, observed thirty-five children who lied between age 5 & 14 ran different doses of ‘vosoritide’.
Those children who got to receive the highest amount of dosage grew at the same rate to the average height children of similar age, an outcome which continued for 3 and a half years.
In the trial, on average the participants grew twice as faster than their standard rate with no negative effects to any proportion of the body.
The leading author of the study, Ravi Savarirayan who is also a Professor of clinical genetics, passed the following statement: “This research is the very first report of a likely incredible, precision therapy for those children who have achondroplasia, which we are hopeful of improving their functioning and health results, as well as, boost their height & access to their surroundings.”
Savarirayan further stated that the results of this ground-breaking drug can have substantial effects on the outcomes of the health of those who are born with this condition. Though he decided not to call the advancement as a cure for achondroplasia. He said: “I would prefer not calling it a cure, rather I would say that on the prospect we have got way better options for the families to evade surgery, to keep their children healthy and prevent them from visiting the hospital.”