This poster, presented at ISPMD 2024, explores the administration of eladocagene exuparvovec using the SmartFlow magnetic resonance (MR)-compatible ventricular cannular in pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency
This symposium, presented at ESGCT 2023, provides an overview of the benefits of gene therapy for patients with aromatic L-amino acid decarboxylase (AADC) deficiency
This infographic, distributed at NSGC 2023, provides an overview of the development of aromatic L-amino acid decarboxylase (AADC) deficiency, as well as the preliminary and core diagnostic tests that should be used for patients with key signs and symptoms
This poster, presented at NSGC 2023, provides an overview of the utilization of artificial intelligence in analyzing Electronic Health Record (EHR) data to identify patients who may be at increased risk of aromatic L-amino acid decarboxylase (AADC) deficiency, with the aim of decreasing diagnostic delay for patients
This infographic, presented at ESGCT 2023, provides an overview of the signs and symptoms of aromatic L-amino acid decarboxylase (AADC) deficiency and the use of gene therapy for treating this rare disease
This oral presentation at AAN 2023 discussed eladocagene exuparvovec, a gene therapy for patients with aromatic L-amino acid decarboxylase (AADC) deficiency, focusing on the efficacy and safety data from three clinical trials
This symposium, presented at SSIEM 2023, provides an overview of the identification and diagnosis of patients with aromatic L-amino acid decarboxylase (AADC) deficiency. It also focuses on the potential of gene therapy as a treatment option
This infographic, distributed at the PTC Therapeutics symposium at SSIEM 2023, gives an overview of the diagnosis and treatment of aromatic L-amino acid decarboxylase (AADC) deficiency
This symposium, presented at ESGCT 2022, provides an overview of gene therapy as a strategy for the management of AADC deficiency
This infographic, distributed during PTC Therapeutics symposium at ESGCT 2022, provides an overview of gene therapy as a strategy for the management of AADC deficiency
This symposium from the 14th EEC Congress, held in Geneva, Switzerland 2022, provides real-world insights into the caregiver and clinician perspective of the diagnosis and management of aromatic L-amino acid decarboxylase (AADC) deficiency
The key findings of a post hoc analysis of the clinical trial results for an investigational gene therapy are discussed in our poster from ISPMD 2022, held in Barcelona, Spain
The key findings of a UK study into the feasibility of a mobile domiciliary phlebotomy service to promote clinical trial recruitment are presented in our poster from ISPMD 2022, held in Barcelona, Spain
View our symposium presented at ISPMD 2022, held in Barcelona, Spain, which discusses the potential role of an investigational gene therapy treatment in the management of AADC deficiency
This infographic, distributed at ISPMD 2022, provides an overview on the rare nature of AADC deficiency
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Access PTC abstracts, infographics, posters and symposia, presented at international meetings in the fields of pediatric neurology, cognitive neuroscience, and neuromuscular disorders, such as the Child Neurological Society (CNS) Annual Meeting, American Society of Gene and Cell Therapy (ASGCT) General Meeting and the International Symposium on Genetic Syndromes with Movement Disorders and Epilepsy (GSMDE).