Tuesday, 29 August 2023
Title: New perspectives in PKU management – The change we can see
12:45 – 13:45
Pincus
Title: AADC Deficiency: Advances in Diagnosis and Treatment
12:45 – 13:45
Dulchin 1
Title: Creating A New Roadmap: Advances in Fabry Disease Monitoring and Management
12:45 – 13:45
Oranim 3 and 4
Wednesday, 30 August 2023
Title: Pharmacological optimization of propionic and methylmalonic acidurias long-term management: the latest evidence from the bench to the bed side
07:30 – 08:30
Dulchin 1
Title: Neurocognitive aspects of Mucopolysaccharidosis II: from early diagnosis to developmental assessment
17:30 – 18:30
Pincus
Title: Pegtibatinase: An Investigational Enzyme Replacement Therapy in Development for Treatment of Classical Homocystinuria
17:30 – 18:30
Oranim 3 and 4
Thursday, 31 August 2023
Title: Treating adult MPS IVA patients: Recent findings from the largest global Morquio A Registry Study
12:30 – 13:30
Pincus
Title: Advancing the Treatment of PKU: Results of the APHENITY Trial
12:30 – 13:30
Dulchin 1
Title: Addressing the needs of patients with Pompe disease – translating clinical trial data into real-world practice
12:30 – 13:30
Oranim 3 and 4