Coordinating Council for Rare Diseases Approves National Achondroplasia Protocol

On July 26, the Coordinating Council for Rare Diseases approved the national protocol on Achondroplasia, as revealed by Council member Ivane Chkhaidze in a statement to journalists.

Chkhaidze expressed satisfaction with the protocol’s reception, highlighting that a German expert highly praised its “comprehensive and informative nature”.

During the meeting, technical aspects concerning the administration of the medication were also addressed. Chkhaidze emphasized that patients will receive their initial dose at the Iashvili Central Children’s Hospital, under close medical supervision, due to potential side effects requiring immediate intervention, particularly a drop in blood pressure.

The number of children in the initial stage was limited to 5-6, with a focus on those aged 11, 12, and 13, as they have the least time remaining to benefit from the drug’s effects, providing them with minimal opportunities for bone nutrition. A multidisciplinary group will oversee the patient selection process, with plans to include other children in subsequent stages.

The council’s decision outlined a program spanning 2023 to 2024, with the participation of the first group of children scheduled to commence before the year’s end, subject to finalizing negotiations with the manufacturer. The treatment will continue for at least a year, with growth progress monitored. If the growth increase during the year falls below 1.6 centimeters, the medication will be discontinued, but successful cases will continue treatment for another year.

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