Foto: Andrea Katheder (https://elhks.de/eva-luise-koehler-forschungspreis2024/)
The scientists were honored for their groundbreaking work on maternally inherited Leigh’s disease (MILS), a rare and severe mitochondrial disease affecting children where treatment options are lacking.
The Federal Minister of Health Prof. Dr. Karl Lauterbach assures political support for research into rare diseases: “Medical progress often does not reach people with rare diseases. This cannot and must not happen!”.
In their SPARK-BIH Project, the team identified a class of drugs applicable for repurposing that restore cellular disease phenotype. The team has initiated compassionate use treatments for terminally ill patients who have recovered significantly after the treatment.
Recently, The European Medicines Agency (EMA) has approved this treatment for MILS therapy in accordance with the Orphan Drug Designation (ODD). A Europe-wide multicenter study for further clinical testing of the active substance is due to start shortly.
More information can be found here: https://elhks.de/en/eva-luise-koehler-forschungspreis2024/
