What are the chances that a girl from Taiwan would marry a man from the U.S., whom she happened to meet in Thailand, and unbeknownst to them, they were both autosomal recessive carriers of an ultrarare disease known as aromatic l-amino acid decarboxylase (AADC) deficiency? Even though…
Rare Disease Day 2022
Recent Posts
- Rare disease workshop focuses on patient-centered drug development
- For families in the AADC deficiency community, there is no perfect path
- SK pharmteco leverages its viral vector expertise in gene therapy partnership
- The endurance competition of keeping up with our early-rising daughter
- AAV2 gene therapy vector movement tracked over time in rat brain study