At the last minute, a desperate search for a home pays off

As my wife, Judy, and I drove through the streets of Taipei on a recent trip to celebrate the journey of the gene therapy Upstaza (eladocagene exuparvovec) in Taiwan, we passed an apartment building I hadn’t thought about in years. A flood of memories came rushing back.

Creating lasting bonds with AADC deficiency families

Once again, my family took a summer trip to Taipei, Taiwan, to contribute data to a clinical trial. Our daughter, Rylae-Ann, is participating in a study of the long-term efficacy of a treatment for aromatic l-amino acid decarboxylase (AADC) deficiency. Our previous trip last summer provided…

AADC Deficiency and Ethnicity

Aromatic l-amino acid decarboxylase (AADC) deficiency is a very rare disease caused by mutations in the gene that encodes for the AADC protein. The disease seems to be more common in some parts of the world, and people of certain ethnicity may be at higher risk for the…

Gene Therapy May One Day Lead to Cure for AADC Deficiency, Review Suggests

A recent report reviewing the use of gene therapy for Taiwanese patients with aromatic l‐amino acid decarboxylase (AADC) deficiency suggests that such strategies may one day lead to a cure for this condition. The report, “A review of aromatic l‐amino acid decarboxylase (AADC) deficiency in Taiwan,” was…