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
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 poster, presented at ISPOR 2022, highlights how the severity of aromatic L-amino acid decarboxylase (AADC) deficiency symptoms may have an impact on the quality of life of both patients and caregivers
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
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
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