This International Day of Persons with Disabilities IDPD know more about Achondroplasia Also find out about its cure ...

"At the moment the only medication approved for the treatment of children with achondroplasia is vosoritide [Voxzogo], which requires a daily subcutaneous injection," Savarirayan told MedPage Today.

The study involved 72 children with achondroplasia from Australia, the UK, the US, Spain, France and Canada. It reported the ...

Achondroplasia is a genetic skeletal condition ... followed by 12 months of extended treatment in which the dose in cohorts 1 and 2 could be escalated to the next ascending level at months 6 ...

Relative to an untreated achondroplasia reference population, the mean change from baseline in height z score was 0.54 at 18 months; in the upper-to-lower body segment ratio, the mean change from ...

Relative to an untreated achondroplasia reference population ... the mean change from baseline was −0.12. "Additional treatment duration will be required to determine whether the improvement ...

This growth effect surpasses the average annualized increase of +1.7 cm/year seen with the daily administration of vosoritide, an approved therapy for achondroplasia. The low-dose cohort comprised 6 ...

A new oral medication, called infigratinib, shows promise in enhancing growth and limb proportion in children with ...

"Furthermore, the breadth of clinical and real-world evidence underscores why we continue to advocate for treatment with VOXZOGO as early as possible." Real-world longitudinal data from the ...

1 According to the NEJM, achondroplasia is a genetic skeletal condition that results in disproportionately ... for 6 months, followed by 12 months of extended treatment in which the dose in cohorts 1 ...