AADC News

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

New AI tool for doctors’ notes may save patients years to be diagnosed
03/12/2025

New AI tool for doctors’ notes may save patients years to be diagnosed

Computer AI analysis of doctors' notes may be an effective screening tool to speed diagnosis of rare diseases, including AADC deficiency.

Computer-guided surgery delivers gene therapy to 2 children
26/11/2025

Computer-guided surgery delivers gene therapy to 2 children

Image-guided surgery safely and accurately delivered the gene therapy Upstaza to two children with AADC deficiency, a study reported.

Patient passports improve life for rare disease families: Study
19/11/2025

Patient passports improve life for rare disease families: Study

Patient passports for rare diseases such as AADC deficiency help patients and caregivers communicate with doctors, a pilot study found.

Gene therapy outcomes in AADC deficiency best tracked by doctors
12/11/2025

Gene therapy outcomes in AADC deficiency best tracked by doctors

Neurotransmitter levels do not predict clinical outcomes after gene therapy in children and adolescents with AADC deficiency, a study found

New mobile app connects rare disease patients to research
05/11/2025

New mobile app connects rare disease patients to research

A new mobile app, IAMRARE, aims to make it easier for people with rare diseases and caregivers to participate in research studies.

NORD summit spotlights how patient experience drives progress
30/10/2025

NORD summit spotlights how patient experience drives progress

The NORD Rare Diseases & Orphan Products Breakthrough Summit highlighted how patient experience drives scientific and policy advances.

Raising awareness with the AADC Family Network this Oct. 23
22/10/2025

Raising awareness with the AADC Family Network this Oct. 23

The AADC Family Network will host its 6th annual AADC Awareness Day event on Thursday, Oct. 23, online and open to the community.

Forum will highlight latest in A*O treatment for ultra-rare diseases
15/10/2025

Forum will highlight latest in A*O treatment for ultra-rare diseases

The N-Lorem Foundation will host its third annual event sharing developments on A*O treatment for nano-rare diseases like AADC deficiency.

Kebilidi gene therapy delivers big quality-of-life boost, study finds
08/10/2025

Kebilidi gene therapy delivers big quality-of-life boost, study finds

Kebilidi, an approved gene therapy for AADC deficiency, delivers greater gains in life quality at lower cost, an analysis shows.

Rare disease patients in 1 US state likely to have delayed diagnosis
01/10/2025

Rare disease patients in 1 US state likely to have delayed diagnosis

Pennsylvania patients with rare diseases like AADC deficiency often face a delayed diagnosis and problems accessing care, a survey found.

FDA introduces process to ease review of rare disease therapies
24/09/2025

FDA introduces process to ease review of rare disease therapies

The FDA has launched a process to ease and speed regulatory review of treatments for rare genetic diseases, like AADC deficiency, in the U.S.

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