News

New European alliance to advance rare disease diagnosis, care

The European Rare Diseases Research Alliance (ERDERA) was launched officially last week with the vision to offer better diagnosis and treatment for the 30 million people living in Europe with a rare disease like aromatic L-amino acid decarboxylase (AADC) deficiency. Coordinated by the National Institute of Health and…

AI model identifies existing drugs to repurpose for rare diseases

An artificial intelligence (AI) model could identify possible therapies for thousands of diseases from existing treatments, according to Harvard Medical School researchers. These diseases include rare disorders such as aromatic I-amino acid decarboxylase (AADC) deficiency, for which there is currently no treatment. The model, called TxGNN, closely resembles the…

OHC symposium focuses on rare disease research, therapy

The Oxford-Harrington Rare Disease Centre (OHC) is hosting its biannual symposium on rare disease therapy development drivers, bringing together experts from academia, biotechnology, pharmaceuticals, venture capital, and patient advocacy to discuss the latest research, therapeutic strategies, and collaboration efforts for rare diseases such as aromatic L-amino acid decarboxylase…