Voxzogo (BMN-111): A Family Dinner Discussion
Voxzogo (BMN-111): A Family Dinner Discussion
What happens when an LP family sits down to discuss a new treatment for achondroplasia — an honest conversation about the questions that matter.
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?
That hypothetical is no longer hypothetical. BioMarin’s targeted pharmaceutical treatment aimed at the FGFR3 gene signaling pathway — known during clinical trials as BMN-111 — has completed that journey. It sparked enormous conversation in our community when it was in development, and it continues to do so now that it is approved and in active use.
📋 Clinical Status: What Parents Need to Know in 2026
BMN-111 is now fully FDA-approved under the brand name Voxzogo (vosoritide), indicated for children with achondroplasia whose growth plates remain open.
- Initial approval: November 2021, for children aged 5 and older
- Expanded approval: October 2023 — now includes children of all ages with open growth plates, including infants and toddlers
- How it works: Daily subcutaneous (under-the-skin) injection, not a pill — administered throughout the child’s active growing years
- Measured outcomes: Over four years, children aged 2 and above showed an average height gain of 6.3 to 7.8 centimeters compared to untreated children with achondroplasia of the same age and sex
- Important note: Like ELL, Voxzogo does not alter the underlying FGFR3 gene — it modulates the signaling pathway. A child treated with Voxzogo will still have achondroplasia and can still pass it to their children.
This post reflects our family’s personal perspective, not medical advice. Always consult a physician experienced with skeletal dysplasias — the LPA Medical Advisory Board is an excellent starting point.
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 of 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 Voxzogo
Because our childhood medical realities were so distinct, Voxzogo’s approval 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, viewing it strictly as a potential 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 not enroll our own children in daily subcutaneous injections throughout their growing years. While we hold absolute empathy and support for families who choose to pursue this 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 pharmaceutical wouldn’t guarantee a happier life.
To the new parents out there looking at modern therapeutic 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.
“If there was a pill to make me average height, would I take it? Only if there was one to turn me back.”
— Renee’s daughter with achondroplasia, via the comments below
Essential Reading for Families
“Dwarfism: Medical and Psychosocial Aspects of Profound Short Stature”
By Betty M. Adelson. The definitive bridge between clinical facts and community culture. Essential for any new parent sorting through a diagnosis.
“Little People: Learning to See the World Through My Daughter’s Eyes”
By Dan Kennedy. An honest, deeply researched look at raising a child with dwarfism — including perspectives on ELL and identity from within the community.
With Voxzogo now available for children of all ages, this conversation has never been more present — or more personal. Where does your family stand? We’d love to hear your perspective, whatever it is, in the comments 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”. 🙂