Satellite Symposia
Participation in the satellite symposia is free of charge. We thank the companies for their programme contributions and support!
Tuesday, 3 September
Sanofi
Transforming ASMD Management: Clinical Perspectives on Timely Diagnosis and Treatment of ASMD
Tuesday, 3 September 2024 | 12:45-13:45
Location:
Camellia Auditorium | Level 0
ULTRAGENYX
Long-chain fatty acid oxidation disorders: from biochemistry to experts’perspectives on diagnosis and management
Tuesday, 3 September 2024 | 12:45-13:45
Location:
Plane Auditorium | Level 0
PTC THERAPEUTICS
Unlocking the potential of patients with AADC deficiency
Tuesday, 3 September 2024 | 12:45-13:45
Location:
Magnolia Auditorium | Level -1
TAKEDA
Can we meet the challenges of living well with Fabry disease?
Tuesday, 3 September 2024 | 12:45-13:45
Location:
Laurel Room | Level -1
Wednesday, 4 September
AMICUS THERAPEUTICS
Navigating multidisciplinary connections in Fabry disease: a patient centered approach from diagnosis to treatment
Wednesday, 4 September 2024 | 07:30-08:30
Location:
Camellia Auditorium | Level 0
CHIESI GLOBAL RARE DISEASES
How to deal with uncertainty in LSDs to improvepatient care. The alpha-mannosidosis case
Wednesday, 4 September 2024 | 07:30-08:30
Location:
Plane Auditorium | Level 0
ImMEDICA PHARMA
Meeting the Unmet Need: Introducing pegzilarginase for the treatment of Arginase 1 Deficiency
Wednesday, 4 Setember 2024 | 07:30-08:30
Location:
Magnolia Auditorium | Level -1
IECURE
Emerging Cell and Gene Therapy Approaches for OTC Deficiency
Wednesday, 4 September 2024 | 07:30-08:30
Location:
Laurel Room | Level -1
Applied Therapeutics
Development of Govorestat (AT-007), the First Potential Treatment for Patients with Classic Galactosemia
Wednesday, 4 September 2024 | 18:30-19:30
Location:
Magnolia Auditorium | Level -1
BIOMARIN
PKU management in evolution: Discussion on the changing soundscape
Wednesday, 4 September 2024 | 18:30-19:30
Location:
Camellia Auditorium | Level 0
Recordati
Optimising care of organic acidurias – Opportunities and challenges when treating patients long-term
Wednesday, 4 September 2024 | 18:30-19:30
Location:
Plane Auditorium | Level 0
Thursday, 5 September
AMICUS THERAPEUTICS
Prioritizing Disease Monitoring in LOPD: Can We Do More?
Thursday, 5 September 2024 | 07:30-08:30
Location:
Camellia Auditorium | Level 0
Jnana Therapeutics
Harnessing SLC6A19 as a potential treatment approach for PKU andrelated amino acid disorders
Thursday, 5 September 2024 | 07:30-08:30
Location:
Plane Auditorium | Level 0
MIRUM PHARMACEUTICS
Future Directions in the Diagnosis and Management of Inherited Metabolic Disorders Affecting Cholesterol Synthesis: Spotlight on CTX and SLOS
Thursday, 5 September 2024 | 07:30-08:30
Location:
Magnolia Auditorium | Level -1
CHIESI GLOBAL RARE DISEASES
Empowering voices in Fabry disease: collaborative advances intreatment and patient care
Thursday, 5 September 2024 | 12:30-13:30
Location:
Camellia Auditorium | Level 0
PTC THERAPEUTICS
Unlocking New Possibilities in PKU
Thursday, 5 September 2024 | 12:30-13:30
Location:
Magnolia Auditorium | Level -1
SANOFI
A Focus on Bone Manifestations in Gaucher Disease
Thursday, 5 September 2024 | 12:30-13:30
Location:
Laurel Room | Level -1
ORCHARD THERAPEUTICS
New Horizons for Metachromatic Leukodystrophy
with the advent of newborn screening
Thursday, 5 September 2024 | 12:30-13:30
Location:
Plane Auditorium | Level 0