I had the honor of being part of this project by the Special Broadcasting Service (SBS), Australia’s most diverse broadcaster, that focuses on the trial for vosoritide, a therapeutic treatment that will soon be available to those affected by achondroplasia.
This SBS Dateline special, Born Small, explores the journey of Casper, a patient of Dr. Ravi Savarirayan at Murdoch Children’s Research Institute, as he goes through the trial for vosoritide.
Check this out: BioMarin Announces New and Updated Data at 2021 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting Demonstrating Commitment to Understanding Achondroplasia and Potential Treatment Choice
It also explores two other journeys of individuals with achondroplasia, mine being one of them. If you know me, you know I’m very passionate about advocating for any & all treatment options for those with achondroplasia. Ten years ago, “any & all” was limb lengthening and limb lengthening alone – aside from surgical treatments that can become an urgent matter for anyone with achondroplasia – shunt, decompression, ear tubes, tracheotomy, spinal fusion, and so on. Did you know in their first 5 years of life, a child with achondroplasia is at 50x GREATER RISK of SIDS than other children? It’s not just being “small, that’s all.”
Just two months after I began my first lengthening in 2010, BioMarin announced their program for BMN-111 for the treatment of achondroplasia. When I was younger, I did wish there were treatment options for me that would not only have prevented a lot of the health risks and complications that come with having achondroplasia but maybe would have made me as uniform as the other girls in my dance class.
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