The EQT Foundation, a branch of the investment organization EQT, will fund breakthrough research grants investigating rare diseases…
Articles by Lila Levinson, PhD
A 3-year-old girl with aromatic l-amino acid decarboxylase (AADC) deficiency successfully received the gene therapy Kebilidi (eladocagene exuparvovec-tneq)…
A $27 million grant will enable Cincinnati Children’s to continue to serve as the hub of a network of rare…
Note: This story was updated July 22, 2025, to correct Nippon Shinyaku (NS)…
More than 70 additional patient organizations have joined a joint statement from the European Organization for Rare Diseases (EURORDIS)…
Researchers in Germany are creating an artificial intelligence (AI) tool to help physicians diagnose rare diseases, such as aromatic…
OneMedNet, a provider of real-world healthcare imaging data, will expand its capabilities to enable secure collaboration for researchers investigating rare…