PUBLISHED JULY 29, 2026

One Step Closer to a Treatment for Little Lora

One year ago, the story was about hope. Today, it is about progress, say the grateful parents of three-year-old Lora from Novo mesto.

With the help of people across Slovenia, they have finally raised €1 million for the development of a treatment that is already progressing in Australia. Due to the rare GAND syndrome, Lora is unable to speak or walk. Only around 500 children worldwide are believed to live with the condition.

Lora’s parents established the GAND Foundation to connect families affected by the same condition around the world and attract international donors. They also hope to receive support from the Slovenian government, which has previously co-funded the development of gene therapy for Urban, a child living with CTNNB1 syndrome.

€1 Million Raised for the Development of Lora’s Treatment

For Rebeka and Jernej, the journey has involved many sleepless nights, doubts about whether they had done enough for their daughter, and the difficult task of repeatedly asking the public for support.

However, they say they are relieved that their efforts were not in vain.

“It is a relief. Together with so many people, we have completed the first enormous part of the journey and raised the €1 million,” said Lora’s mother, Rebeka Marolt.

The funds covered the initial development of the treatment for GAND syndrome, which is progressing well.

“The vector that will deliver the gene into the cell has already been developed. We are now in the phase of developing the mouse model. We have a control group and a group carrying the altered gene that Lora has, meaning the mice have GAND syndrome. The study in mice will now begin,” explained Lora’s father, Jernej Blatnik.

After the treatment’s safety and effectiveness have been evaluated, the next steps will include the costly production of the therapy and a clinical trial.

Because of the uncertainty involved in gene therapy development, the family did not initially want to raise money for these later stages in advance.

“We estimate that an additional €3 million will be required to complete the entire process,” the parents said.

Establishing the GAND Foundation

To complete the project together with other families affected by GAND syndrome, Rebeka and Jernej established the GAND Foundation.

They also hope that the Slovenian government will support the project.

“Under the legislation adopted in recent years, the government could contribute up to €1 million for Lora,” Rebeka said.

Although the family no longer wishes to actively ask the public for donations, Jernej explained that people who feel connected to the story may still contribute through the available donation channels.

Lora will celebrate her fourth birthday in September. The sooner she receives the treatment, or the progression of the condition is stopped, the greater the chance of reducing its long-term effects.

“According to the current timeline, we hope this could happen at the beginning of 2028,” said Jernej.

The progress of Urban, who received treatment for CTNNB1 syndrome and is now walking and speaking seven months later, gives the family additional hope.

“I think Urban is like a guiding star, leading Lora forward,” Jernej said.

Author: Tjaša Dugulin
Original source: 24UR
Original title: Še korak bližje do zdravila za malo Loro
Published: 25 July 2026

This article is based on reporting originally published by 24UR Slovenia. Read the original article here.