Casper is an energetic five-year-old born with achondroplasia, the most common form of skeletal dysplasia. Most recently, he’s turned everyone’s head by participating in a groundbreaking clinical trial for the new drug vosoritide. Quickly this treatment has become the focal point of heated debate between parents and medical practitioners. They are laying the groundwork to effectively and ethically argue against interventions for children with dwarfism at all. His journey began at the miraculous age of just five months when he enrolled in a clinical trial. From that young age, he’s experienced important milestones and challenges, all with the support of a caring community.
Casper’s parents had to deal with enough advocates’ fears about the treatment itself, as well as its long-term consequences. When he was 17 months old, they got a wonderful call. He’d still enroll in a long-term study at Melbourne’s Murdoch Children’s Research Institute, the largest clinical trial for vosoritide in the world. This announcement came as a wave of hope and trepidation for his family.
Over the years, Casper has had a few small health problems related to his condition. His parents, though, are learning that these challenges are not insurmountable. Perhaps even more significantly, he recently took his first steps independently. This achievement has brought both pride and elation for Basil’s parents. His mother, Daisy, shared her feelings about his progress, saying that she feels “really proud of the progress that he was making.”
Casper is frequently referred to as a “cute athletic little sporty boy. To aid in his mobility on land, he ambulated with arm crutches. She brings him out into the community, works with him on all kinds of day-to-day activities. In April, Casper sprinted down the streets of Harvard Square, Massachusetts. His spirit was truly indomitable, as it had to be, supported by the indomitable spirit of his community.
Despite facing societal challenges, Casper and his family have found camaraderie in groups such as the Short Statured People of Australia. These meetups offer an essential arena for families like theirs to find and interact with other families who’ve walked such paths. Daisy noted the importance of these interactions, saying, “There’s short-statured adults as well in there, which is awesome because you can sort of bounce ideas off of them.”
Casper’s medical team has not been clear with his family about the realities of achondroplasia. They provided “really sobering statistics on what life might look like for Casper,” prompting his parents to reflect on their approach to the treatment. While they were once just debating if they should seek vosoritide treatment for Casper, now their efforts are directed at maximizing his long-term independence.
Daisy emphasizes the value of community support and shares her appreciation for the diversity of experiences among families attending these meetups. She remarked, “You get the opportunity to speak to parents who’ve got older children who have been there, done that. So it is a really great supportive community for sure.”
Deciding to enroll Casper in the clinical trial wasn’t a decision made lightly. The family wrestled internally with questions about why they were doing this and what this would entail for Casper’s future. Becky, an advocate for individuals with dwarfism, posed important questions about societal perceptions and parental motivations: “Is it the family that doesn’t feel confident about their child’s future that’s trying to fix them?”
Amidst these reflections, Becky remains hopeful about raising awareness regarding treatment options while acknowledging the importance of individual choice. She stated, “At the end of the day, it’s your decision, but there is so much positivity out there, even if you don’t pursue that route. There are possibilities.”
As Casper goes deeper down this path, his family is still very much concerned with celebrating what Casper can do and encouraging his independence. Daisy expressed her pride in being Casper’s mother, stating, “At the risk of sounding like a really biased, proud mum, I am loving every moment of being Casper’s mum.”
