As far as I know, Vosoritide is the only official medicine available to date. Below is the summary of its features
・The first approved medicine in the world for Achondroplasia(ACH)
・Its effect is to help increase their height by inhibiting the activity of FGFR3,
which act as a negative regulator for bone growth. ・It is not a tablet; it requires a daily subcutaneous injection ・You must keep it in the refrigerator.
【How is it working for my daughter?】
My daughter was prescribed it three years ago. We can see that it is actually helping her bones grow (as shown in the diagram). The X-axis represents age, while the Y-axis shows her height. Until she was 5, her growth followed the average curve, but since then, the slope has become steeper, showing that the medication is working well. It adds about 1.5 cm of height increase per year in addition to natural growth. Regarding the disproportion of her arms and legs, it seems to be helping a bit—at least it is preventing it from worsening. Please be aware that its effects vary from person to person, so please take these results as a reference only.
【How is it managed every day?】
They are stored in our refrireirator, taking up quite a bit of spaces. The medicine itself comes in powder form. After mixing it with diluent, you need to draw it into the syringe. The manual says that recommened injection place is either arms, thigh,abdomen or bottucks. We are injecting it to her buttocks. It was tough for her to get injected as she was only 4 years old when it was started. My wife and I had to hold her down in order to prevent her from moving. It is dangerous to let her move as the needle is in her buttocks. We do this every single day. When we travel, we bring the medication in a cooler bag with ice, which takes extra time and effort.
【Are there other medicines out there】?
I know there are other medications currently undergoing the regulatory approval process overseas (US and EU). With these, injections would only be required once a week. This would significantly reduce the daily burden on families. We hope they will be approved and released at many countries.
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