AADC News

AADC News We are dedicated to sharing the latest news, research, and patient perspectives from the AADC community.

Tube feeding linked to poorer oral health in children: Study
28/08/2025

Tube feeding linked to poorer oral health in children: Study

Children with rare diseases who require a feeding tube inserted into the stomach tend to have poorer oral health, a study found.

Texas hospital uses Kebilidi gene therapy to treat 3-year-old girl
21/08/2025

Texas hospital uses Kebilidi gene therapy to treat 3-year-old girl

Kebilidi, which is sold as Upstaza in Europe, became the first approved gene therapy for AADC deficiency in the U.S. last year.

Texas hospital uses Kebilidi gene therapy to treat 3-year-old girl
20/08/2025

Texas hospital uses Kebilidi gene therapy to treat 3-year-old girl

Kebilidi, which is sold as Upstaza in Europe, became the first approved gene therapy for AADC deficiency in the U.S. last year.

Grant boosts rare disease research role for Cincinnati Children’s
13/08/2025

Grant boosts rare disease research role for Cincinnati Children’s

Cincinnati Children’s received $27 million to extend its role as the coordinating hub for a rare disease research network for five years.

New cell model hailed as platform for studying AADC deficiency
07/08/2025

New cell model hailed as platform for studying AADC deficiency

A recently developed cell model is being called “a significant step toward understanding the molecular mechanisms of AADC deficiency.”

Study emphasizes need for holistic support for rare disease patients
30/07/2025

Study emphasizes need for holistic support for rare disease patients

Patients affected by rare diseases like AADC deficiency need holistic support, not just medical care, per a review of over 250 studies.

Girl with AADC deficiency thriving after Kebilidi treatment
23/07/2025

Girl with AADC deficiency thriving after Kebilidi treatment

A 3-year-old girl with AADC deficiency treated with Kebilidi at Boston Children’s Hospital as a baby is now thriving, doctors said.

Nippon Shinyaku, Boston Children’s to partner on rare disease research
22/07/2025

Nippon Shinyaku, Boston Children’s to partner on rare disease research

Nippon Shinyaku Pharma will partner with Boston Children’s Hospital to study and develop treatments for rare diseases like AADC deficiency.

Nippon Shinyaku, Boston Children’s to partner on rare disease research
22/07/2025

Nippon Shinyaku, Boston Children’s to partner on rare disease research

Nippon Shinyaku Pharma will partner with Boston Children’s Hospital to study and develop treatments for rare diseases like AADC deficiency.

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