Slovenia will co-finance the final stages of research of orphan drugs to help children with rare diseases under legislative amendments confirmed by parliament after the case of a five-year-old boy with a rare genetic disorder prompted a massive fundraising campaign.

Under the changes to the Scientific Research and Innovation Activity Act confirmed on 20 November, applicants will be able to petition the government to finance drug development based on a positive opinion from the Agency for Medicinal Products and Medical Devices.

Financing will be limited to one million euros per drug and to drugs being developed by Slovenian organisations, which can collaborate with international organisations.

The legislation was fast-tracked through parliament to provide the funding for the development of a treatment for the CTNNB1 syndrome, which is spearheaded by a Slovenian researcher, a mother whose son suffers from the syndrome.

The CTNNB1 syndrome is a severe neurodevelopmental disorder caused by a mutation at the chromosome 3p22.1 of the CTNNB1 gene, which affects about one in 50,000 children. It causes delays in development, intellectual disorders and speech delay.

Since the condition is so rare, the pharma industry is not interested in funding the expensive research, so Urban's mother, Špela Miroševič, a researcher at the Medical Faculty of the University of Ljubljana, managed to bring together a team of scientists from all over the world to develop a gene therapy.

She has also founded the CTNNB1 Foundation, which has raised more than €2 million for the project in a two years in collaboration with a Slovenian charity.

The therapy has passed pre-clinical testing and another €250,000 is reportedly needed for the final phase of development.