Connect with a community of support
CRYSVITA Educational Events for Patients and Caregivers
The CRYSVITA Educational Events program offers events that are presented by an XLH specialist. These events provide opportunities to learn more about CRYSVITA and XLH and connect with local members in the XLH community.
XLH Network
The XLH Network is a global patient support organization for people with XLH and their families and caregivers. The website contains educational resources about XLH for patients and medical professionals.
NIH Genetic and Rare Diseases Information Center (GARD)
GARD maintains a list of rare diseases and related terms to help people find reliable information about their disease.
National Organization for Rare Disorders (NORD)
NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them.
Global Genes
Global Genes is a rare disease patient advocacy organization that works to build awareness, educate the global community, and provide connection and resources.
GLOSSARY
antibody
A protein that is made by the immune system or a protein that is modified in a lab to recognize and block a target that threatens the body’s normal function.
baseline
The beginning of a study before patients are given any treatment.
conventional therapy
A therapeutic approach for XLH that consists of oral phosphate and a type of vitamin D called active vitamin D.
fibroblast growth factor 23 (FGF23)
A protein that plays a role balancing the amount of phosphorus in the body; in XLH, the activity of FGF23 is abnormally increased, throwing off the balance of phosphorus.
fractures
Broken bones.
least squares (LS) mean
A statistic that estimates the mean after adjusting for the effect of other variables in a study.
mineralization
A process in which minerals like phosphorus are deposited in the bone, causing the bone to harden.
osteoid volume to bone volume (OV/BV)
A ratio of unmineralized bone to mineralized bone; in XLH, this ratio is large, pointing to a greater presence of unmineralized bone.
osteomalacia
A softening and weakening of the bones in children and adults caused by deficiencies in vitamin D, calcium, or phosphate, independent of the growth plate, which allows the bones to bend easily.
phosphorus
A mineral that is important for proper bone development and repair.
placebo
A harmless pill, medicine, or procedure that has no therapeutic effect; in Study 4 of adults with XLH, placebo refers to a saline injection.
pseudofracture
A kind of fracture where the bone thickens at the site of an injury but is not completely broken.
Radiographic Global Impression of Change (RGI-C)
A way to measure the extent of the healing of rickets. This score uses a 7-point scale to rank the healing of rickets and can range from -3=severe worsening to 0=no change to +3=near/complete healing. An RGI-C global score of ≥+2.00 means a substantial healing of rickets was achieved after treatment.
rickets
A softening and weakening of the bones in children caused by deficiencies in vitamin D, calcium, or phosphate before the growth plate closes.
serum phosphorus
Amount of phosphorus in the blood.
Thacher Rickets Severity Score (RSS)
A way to measure changes in the severity of rickets. This score uses a 10-point scale to rank the severity of rickets from 0 (normal) to 10 points (severe). A lower score means reduced severity of rickets. This score is complementary to RGI-C.
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