AADC News

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

Rare disease workshop focuses on patient-centered drug development
27/05/2026

Rare disease workshop focuses on patient-centered drug development

EveryLife Foundation’s Scientific Workshop explored patient-centered ways to improve rare disease therapy development and treatment reviews.

SK pharmteco leverages its viral vector expertise in gene therapy partnership
20/05/2026

SK pharmteco leverages its viral vector expertise in gene therapy partnership

The effort aims to develop viral vectors to deliver genetic material into cells to treat blood and metabolic disorders like AADC deficiency.

AAV2 gene therapy vector movement tracked over time in rat brain study
13/05/2026

AAV2 gene therapy vector movement tracked over time in rat brain study

Scientists tracked how an AAV2 gene therapy vector similar to the one used in Kebilidi moves through the brain in rats.

NORD teams with AI company to create rare disease resources
06/05/2026

NORD teams with AI company to create rare disease resources

NORD is teaming up with the artificial intelligence company Openevidence on a project to create resources for thousands of rare diseases.

Natera launches Zenith genomics to help speed rare disease diagnosis
29/04/2026

Natera launches Zenith genomics to help speed rare disease diagnosis

Natera launched Zenith genomics, a genetic testing platform designed to significantly improve rare disease detection.

New AI project aims to solve mysteries of rare childhood diseases
22/04/2026

New AI project aims to solve mysteries of rare childhood diseases

A new project will leverage genomic data from 100,000 children to better understand the biological underpinnings of rare childhood diseases.

New ‘zero-shot’ AI approach aims to jumpstart rare disease treatments
15/04/2026

New ‘zero-shot’ AI approach aims to jumpstart rare disease treatments

First Atom’s new zero-shot AI-powered platform aims to develop novel treatments for rare diseases like AADC deficiency.

USC team develops new AI model to advance rare disease therapies
08/04/2026

USC team develops new AI model to advance rare disease therapies

USC researchers received up to $6.8 million to develop an AI model to advance rare disease therapies in children, including AADC deficiency.

Gene therapy helps children with AADC deficiency gain movement skills
02/04/2026

Gene therapy helps children with AADC deficiency gain movement skills

One-time gene therapy helped children with AADC deficiency gain movement skills, with greater improvements in younger patients.

California’s CIRM aims to speed therapies’ move from lab to clinic
11/02/2026

California’s CIRM aims to speed therapies’ move from lab to clinic

California's CIRM aims to speed the development of gene therapies for rare diseases, funding a $100 million plan to create a model.

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