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
- Pact to develop 3D models could help speed new rare disease treatments
- The power of play: How game night became our daughter’s best therapy
- MRI-guided gene therapy targets serotonin in AADC deficiency
- An ice park may have been a tourist trap, but it was also a therapy win
- New platform aims for personalized gene editing for rare diseases