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
- FDA unveils plan to speed personalized treatments for ultra-rare diseases
- Learning about rare disease through education and by sharing our own story
- Access to care for rare diseases still varies by state: NORD report card
- A long day of play reminds us why a child’s sleep schedule is important
- Digital clinical measures launched to support rare pediatric disease trials