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
- Helpful strategies to mitigate our child’s haircut anxiety
- Planning for the summer heat for your child with AADC deficiency
- Specific gene pairings alter the severity of AADC deficiency cases
- There was no road map for what to do after gene therapy for AADC deficiency
- Looking back at a key developmental milestone following gene therapy