So, there's this drug…
It’s a question I’ve asked myself since learning of Mira’s diagnosis: If a medication could make her taller, would we give it to her?
Recently, this question went from being a hypothetical one to a reality.
On November 19th 2021, The Food and Drug Administration approved the first ever treatment for the most common cause of dwarfism. The treatment is a once-daily injection for children 5 and older with achondroplasia whose growth plates are still open, meaning they still have the potential to grow.
Voxzogo, developed by BioMarin Pharmaceutical, is designed to counteract the genetic mutation that causes achondroplasia. For people with achondroplasia, the natural process by which cartilage turns into bone is interrupted, resulting in shorter stature and the risk of serious medical complications, some of which, Mira has already experienced.
In the clinical trial, patients who received the drug, grew on average 1.6 centimeters more over the course of a year than those who received placebo, with minimal side effects. If taken over many years (likely 10-12), the drug could produce a significant increase in height. For Mira this could mean the difference between reaching a projected adult height of 4 feet tall, or be given an additional 6 inches, maybe more.
This treatment could enable Mira to reach the sink in a public restroom, ride a bike or drive a car without multiple adaptations. She could experience the thrill of a rollercoaster, and buy a dress right off the rack.
The world would literally be within her reach.
More importantly, increased growth would suggest improvements for many of the complications that arise when that growth is inhibited.
Of course, like many medical interventions, not everyone was pleased with the approval.
Some are calling the focus on growth "a pharmaceutical solution for a societal problem."
Some say parents are playing God.
Some say you are sending the wrong message to your child where being a little person is a unique trait to be celebrated, not a problem or a disease in need of a cure.
As for Evan and I, we don’t know a parent that wouldn’t want the very best chance for their child to live a happy, healthy life, free from as many challenges as possible.
This is quite possibly the most difficult decision Evan and I will make in our lifetime. Mira may be grateful for it, but deep down would she think that we rejected who she was? Or worse, would she resent us for not giving her the treatment? This carries a heavy emotional challenge for all of us. Her doctors, while fully supportive of the safety and efficacy of the treatment, are most concerned with the toll this will take on Mira as she grows up.
BioMarin estimates that 21,000 children worldwide — about 3,000 of them in the U.S.— have achondroplasia and are eligible for treatment with Voxzogo.
Mira is one of these children and we are her parents and there’s this drug…