Abbreviations

1,25(OH)₂D: 1,25-dihydroxyvitamin D

25(OH)D: 25-hydroxyvitamin D

6MWT: 6-minute walk test

AE: adverse event

ALP: alkaline phosphatase

BALP: bone-specific alkaline phosphatase

BFI: brief fatigue inventory

BLA: biologics license application

BPI: brief pain inventory

CI: confidence interval

CMA: conditional marketing authorisation

CTx: carboxy-terminal cross-linked telopeptide of type 1 collagen

ECG: electrocardiogram

ECHO: echocardiogram

EMA: European Medicines Agency

FDA: Food and Drug Administration

FGF23: fibroblast growth factor 23

GEE: generalised estimating equation

GLMM: generalised linear mixed model

iPTH: intact parathyroid hormone

LLN: lower limit of normal

LS: least squares

MAA: marketing authorisation application

MAR: mineral apposition rate

MS/BS: mineralising surface/bone surface

MS/OS: mineralising surface/osteoid surface

NR: not reported

NS: non significant

P1NP: procollagen type 1 N-terminal propeptide

PFx: pseudofractures

PHEX: phosphate-regulating endopeptidase homolog on the X chromosome

PTH: parathyroid hormone

Q2W: every two weeks

Q4W: every four weeks

RGI-C: radiographic global impression of change

ROW: rest of the world

RSS: rickets severity score

SC: subcutaneous

SD: standard deviation

SE: standard error

TEAEs: treatment-emergent adverse events

TmP/GFR: ratio of the renal tubular maximum reabsorption rate of phosphate (TmP) to glomerular filtration rate (GFR).

ULN: upper limit of normal

WOMAC: Western Ontario and McMaster Universities Osteoarthritis Index

XLH: X-linked hypophosphataemia