Recently-characterized mutations that have been associated with mild AADC deficiency cause some alterations to the AADC protein, but do…
Articles by Marisa Wexler, MS
A viral vector-delivered gene therapy can restore the activity of the AADC enzyme in people with AADC deficiency for…
The investigational gene therapy PTC-AADC improved motor function in children with aromatic l-amino acid decarboxylase (AADC) deficiency in…
Researchers have developed a patient-derived cell model of aromatic l-amino acid decarboxylase (AADC) deficiency that could provide important insights…
A new online resource, called AADC Insights, aims to make it easier for healthcare professionals to diagnose and care…
The science behind viral gene therapy — which may hold a potential cure in a single treatment for genetic diseases…
Screening genomic databases for disease-causing mutations could help identify people with possible aromatic l-amino acid decarboxylase (AADC) deficiency or…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
Massachusetts is holding its first-ever AADC Deficiency Awareness Day on Oct. 23, according to a press release from…