Young boy with dwarfism given transformative medication to support his growth and height increase

The aspiration of a 13-year-old boy to match his friends’ pace on the soccer field might soon come true, as he has been granted the opportunity to access revolutionary medication for dwarfism at a Sydney hospital. Individuals with achondroplasia, a prevalent form of dwarfism, can now avail themselves of the recently subsidized medication named Vosoritide at The Children’s Hospital at Westmead, with the aim of enhancing their height.

Riley Schroder holds the distinction of being the inaugural recipient of this medication and holds optimistic expectations that it will empower him to pursue his sports-related aspirations.

“I aimed to increase my height and participate on the larger soccer field alongside my friends,” shared Riley.
His mother, Lousie Martin, was deeply moved by the advancement in medical treatment.
“It brought me to tears—tears of joy,” expressed Martin.

According to her, this medication would facilitate his ability to access higher objects, potentially enable him to drive, increase his employment prospects, and address additional health concerns.

“Riley is optimistic about discontinuing the use of his CPAP machine, which he has relied on since birth,” Martin added.

Minna Koo, a physician at The Children’s Hospital at Westmead, explained that the sole previous option involved undergoing limb-lengthening surgery, a protracted and painful procedure. The condition arises due to a mutation in the FGFR3 gene, which hinders bone growth.

Vosoritide operates on a receptor to dampen this activity and stimulate growth. It has the potential to increase the growth rate from a yearly three centimeters to six centimeters.

“Depending on when the treatment begins, these children’s projected adult height has been elevated by at least 10 centimeters,” Koo elucidated. “The results for these youngsters have been nothing short of astonishing.”

Administered on a daily basis, the treatment is well-tolerated and directly influences the growth plates in the bones. It is exclusively suitable for children still in their growth phase, aged between two and 18 years old. Koo noted that the decision is a personal one for each child and family.

Over time, its effectiveness in averting lasting complications like breathing and hearing issues, or the necessity for spinal surgery, will become evident. The drug’s inclusion on the Pharmaceutical Benefits Scheme (PBS) and the subsequent benefit for around 300 children, including Riley, were made possible by international trials led by medical professionals at Westmead and the Murdoch Children’s Research Institute.