Voxzogo (BMN-111): A Family Dinner Discussion
What if you could take a pill to make your achondroplasia go away?
Imagine a reality with no further risk of spinal or foramen magnum compression. A reality where the chances of central and obstructive sleep apnea drop drastically, and where you no longer face the bothersome task of hunting down a specialized bicycle helmet to safely fit a larger cranial profile.
Would you take that pill? If it promised to lessen your child’s chances of navigating social hurdles and physical medical struggles, would you consider administering it to them?
BioMarin has been in the process of developing a targeted pharmaceutical treatment aimed at altering the signaling pathway of the mutated FGFR3 gene completely. Known in clinical trials as BMN-111, this development has sparked immense conversation across our community.
The investigational drug BMN-111 has since transitioned out of trials and is fully FDA-approved under the brand name Voxzogo (vosoritide). It is indicated for pediatric patients with achondroplasia whose growth plates remain open to increase linear height growth. Rather than a simple pill, the treatment is administered via a daily subcutaneous (under-the-skin) injection throughout the child’s active growing years.
Medical Research vs. “Curing” an Identity
Our family actively participates in clinical research tracking sessions during the annual Little People of America (LPA) National Conferences. We readily volunteer for medical diagnostic clinics—measuring everything from blood pressure baselines and body mass index to advanced audiology tracks. My husband and I even turn it into a lighthearted competition to see who clocks the healthiest vitals. However, our motivation is to help physicians establish accurate, safe clinical guidelines to better care for the next generation, not to eradicate our physical traits.
I have lost several deeply cherished family members and close friends to cancer. That, in my definitive opinion, is a horrific disease demanding an absolute cure. My short stature, my snoring patterns, my distinct facial structure, my spinal stenosis, and the societal discrimination I face as a little person do not represent a medical condition waiting to be cured.
The Google Fear Factor for New Parents
If you pick up standard medical journals detailing skeletal dysplasias, you will quickly find descriptions detailing the most severe, dismal clinical outcomes imaginable. When new average-height parents (POLPs) first receive a diagnosis and search “Achondroplasia” online, they are immediately flooded with these clinical horror stories. Terrified and overwhelmed, they search the internet looking for a single glimmer of hope.
As miraculous as the internet is for instantly linking families, it can easily drown you in worst-case diagnostic statistics. If you look instead to adult LPs who have lived the experience, you will discover a completely different reality. Our daily lives are just as normal, diverse, and variable as any pair of high school classmates. Above all else, we learn to adapt, innovate, and thrive completely in a world built for typical heights.
Two Separate Journeys with Dwarfism
Because my husband has pseudoachondroplasia and I have achondroplasia, our early childhood medical paths looked incredibly different. When I was born in the mid-1970s, there were no prenatal ultrasound indications of dwarfism, yet the delivery room staff recognized my physical presentation immediately. My parents got me involved with the Little People of America organization by three months of age, and I began seeing specialized skeletal dysplasia pioneer Dr. David Rimoin at UCLA Harbor-General for my annual evaluations.
Growing up, my parents took me to see regional specialists regularly. I experienced the typical emotional waves of a short-stature child born to average-height parents: wondering “Why me?”, wishing I was taller, and feeling uncomfortable with public staring. Yet, my support network was unshakeable. I was raised to believe there were no structural limits on what I could achieve.
Physically, my pediatric health track was largely smooth. I needed glasses by third grade, orthodontic extractions and braces at twelve, and had mild sleep apnea at fourteen that resolved completely following a standard tonsillectomy and adenoidectomy. For the most part, my childhood was unmarred by severe medical issues, a reality I attribute to a mix of luck, faith, and early community advocacy.
My husband’s introduction to dwarfism was vastly different. His pseudoachondroplasia was diagnosed at age three by an astute pediatrician who cross-referenced his hand X-rays with a medical textbook when his height began dropping off standard pediatric growth charts. He didn’t connect with specialty clinics or Dr. Rimoin’s team until age eight, when orthopedic complications began to manifest.
By the time he reached the eighth grade, he had already undergone an osteotomy on both legs to correct severe bone bowing, alongside a major spinal surgery to correct kyphosis. He navigated two separate rounds of intense corrective orthodontics. Yet, despite major surgeries and substantial recovery downtime, he excelled academically and threw himself into student leadership.
Our Family’s Take on BMN-111 (Voxzogo)
Because our childhood medical realities were so distinct, BioMarin’s pharmaceutical pipeline sparked incredibly mixed feelings at our dinner table. (Then again, we can rarely agree on a standard grocery shopping list!)
If you ask me directly, I would never personally choose to take a pharmaceutical agent to alter my dwarfism. Medical advancements and genetic modifications can never guarantee a smooth or perfect life. My identity, my culture, and my self-worth are deeply rooted in my journey as a little person.
My husband leans more open-minded toward the clinical concept of BMN-111, viewing it strictly as a potential pharmaceutical tool to help future children avoid the severe orthopedic pain, bowing, and spinal surgeries that marked his own youth. However, we both agree that we would never enroll our own children in experimental drug trials or subject them to years of daily subcutaneous hormone injections while their growth plates are fusing. While we hold absolute empathy and support for families who choose to pursue this pharmaceutical path, it simply isn’t the right choice for our household.
Dwarf Identity is Worth Keeping
Ultimately, our lives are full, successful, and beautiful. We have earned degrees, built thriving professional careers, and raised a fantastic family. Yes, we navigate occasional skeletal issues, but changing our physical framework via a magic pill wouldn’t guarantee a happier life.
To the new parents out there looking at modern therapeutic drug pipelines with immense anxiety: take a deep breath. It is completely okay for your child to keep their dwarf identity. They can—and will—live a deeply joyful, successful, and perfectly complete life exactly as they are.
Essential Guide for New Parents: “Dwarfism: Medical and Psychosocial Aspects of Profound Short Stature”
By Betty M. Adelson
If you are a parent trying to sort through clinical literature while understanding the beautiful community culture your child is entering, this book is an absolute must-read. It masterfully bridges medical facts with real-world sociological insights.
Where does your family stand on the choice between pharmaceutical growth therapies and celebrating a natural short-stature identity? Let’s keep this important, respectful dialogue going in our community comments section below.
Awesome post. I love learning from you and your family and your husband’s experiences as well.
Thank you for sharing your thoughts so openly and profoundly. <3
Glad to gain your insight. Most of the Pharma sites are not even bothering to address that there are humans with emotions involved, but focusing on the “several hundred thousand dollar a year potential costs and profits”. Many of the sites also refer to the dramatic increase in BioMarin’s stock value but not one gave any information about patients. I’ve never looked on Achrondroplasia as a curse that had to be eliminated at all costs for the good of society but that was the thrust of these articles. I have a good friend who had rapidly lost his eyesight during high school and by the time I met him could only differentiate between the brightest lights and total darkness hence he never had any I idea what I look like but judged me solely on conversations, I think about that a lot when I first meet people.
I once asked my daughter with achondroplasia if there was a pill to make her average height, would she take it. Her response “only if there was one to turn me back”. 🙂