The recommended starting dose of Crysvita (burosumab) is too low to effectively manage X-linked hypophosphatemia (XLH) in children with the rare inherited disorder, a Swedish study indicates. That dose, of 0.4 mg/kg, is insufficient to normalize the phosphate levels in the bodies of children with XLH, according to…

One-year treatment with Crysvita (burosumab) reduced bone damage and increased phosphate levels in children and adolescents with X-linked hypophosphatemia (XLH), a study showed. The study, “Real-life clinical study: 1-year of treatment with burosumab of children and adolescents affected with X-linked hypophosphatemia,” was presented online at…

If you have a rare genetic disease like X-linked hypophosphatemia (XLH), and are considering whether to have a baby, you may wonder if your children will inherit the progressive disorder. You and your partner should discuss this possibility with your doctor, who may recommend genetic testing. What causes…

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s son, Maximilian, has cystic fibrosis (CF), making him particularly susceptible to COVID-19 and the complications that can arise from it.

Novel mutations in the PHEX gene, the underlying cause of X-linked hypophosphatemia (XLH), do not aggravate the severity of the disease in children, a study from China shows. Certain mutations, however, affected trafficking of the PHEX protein inside cells, supporting the role of protein location on disease development…

One-year treatment with Crysvita (burosumab) led to the normalization of phosphate levels in children with X-linked hypophosphatemia (XLH) and helped improve their growth, a small real-world study shows. The findings provide evidence of Crysvita’s effectiveness beyond clinical trials, and show that the therapy has positive effects without…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

Approximately one third of children and young adults with X‐linked hypophosphatemia (XLH) are overweight or obese, a study has found. Lack of family history of XLH and longer treatment with phosphorus supplements were identified as risk factors for excessive weight gain in these patients. The study, “…

The U.S. Food and Drug Administration (FDA) approved a label update for Crysvita (burosumab) to cover treatment of X-linked hypophosphatemia (XLH) for patients 6 months and older. This update includes data showing improvement in stiffness, continued healing of fractures, and maintenance of efficacy with longer-term use of Crysvita…