Achondroplasia and Therapy: Experiences, Results, and What Comes Next?

On Friday, March 14, 2025, a significant event took place at Belgrade’s Media Center — the second national roundtable on achondroplasia, a rare condition that affects bone growth and causes short stature. The event, organized by the Association “Children with Achondroplasia of Serbia,” brought together experts, parents, doctors, and government representatives to discuss what is working, what has been learned, and what is next for children undergoing therapy in Serbia.

What Is Achondroplasia?

Achondroplasia is a rare genetic condition that affects the growth of bones, particularly in the arms and legs. Children with achondroplasia are shorter than other children their age, and they may face health challenges like back pain, breathing problems, or difficulty moving. But there is now a therapy that can help — and that’s what this meeting was all about.

A Look Back — and a Big Step Forward

The first patients in Serbia started treatment in March 2023. Two years later, eight children are receiving therapy, and their progress is being closely monitored. The results so far are encouraging, and more children are expected to join the treatment program in 2025.

The roundtable discussions covered many important topics, including:

• Real-life stories from the clinic

• How therapy is helping children grow and feel better

• What doctors have learned over the past two years

• Who can start therapy, and how decisions are made

Round table organized by "Children with achondroplasia in Serbia"

Who Was There?

Representatives from Serbia’s Ministry for Family and Child Welfare and the National Health Insurance Fund attended, along with leading medical experts in clinical genetics, such as:

• Prof. Dr. Goran Čuturilo and Dr. Marija Mijović from the University Children's Hospital “Tiršova” in Belgrade

• Dr. Tatjana Stanković from the University Clinical Center Niš

These experts shared updates from their work with patients and discussed how doctors track growth, utilizing tools such as X-rays and charts to monitor children's development.

Therapy Means More Than Just Getting Taller

One of the most powerful messages from the roundtable was this:
Therapy isn’t just about height — it helps the whole body. The doctors explained that the treatment improves how children move, breathe, and live their daily lives. It can also reduce the likelihood of future health issues and enhance overall quality of life.

Government Support and a Promising Future

Representatives from the Health Insurance Fund stated that they are committed to continuing their support for this therapy by funding new patients. The Ministry also announced new plans to hear directly from families about their needs and to further develop the Rare Disease Registry, which already includes over 14,000 patients across Serbia.

“See You Again Soon” — Not “Goodbye”

The event ended on a hopeful note, with participants saying, “See you again,” instead of “Goodbye.” Everyone agreed that this conversation is far from over. As new results come in and more children begin therapy, the dialogue will continue, with one goal in mind: to make life better for children with achondroplasia and their families.