12.00-12.30 GMT
Sponsored Satellite Symposium 1:
Glycomacropeptide (GMP) and Bone Health
Chair: Peter Freisinger (Reutlingen, Germany)
Speaker: Anne Daly (Birmingham, United Kingdom)
Sponsored by
Sponsored Satellite Symposium 2:
Long-Chain Fatty Acid Oxidation Disorders: Evolving Best Practices
Welcome and Introduction
Jerry Vockley (Pittsburgh, United States)
LC-FAODs: Is Guideline-Based Management Enough?
Jerry Vockley (Pittsburgh, United States)
Barbara K. Burton (Chicago, United States)
Ute Spiekerkötter (Freiburg, Germany)
What Will Emerging Treatments Mean for LC-FAOD Management?
Jerry Vockley (Pittsburgh, United States)
Barbara K. Burton (Chicago, United States)
Ute Spiekerkötter (Freiburg, Germany)
Concluding Remarks
Jerry Vockley (Pittsburgh, United States)
Patients with long-chain fatty acid oxidation disorders (LC-FAODs) often experience significant metabolic events leading to emergency room visits, hospitalizations and even mortality, all despite following guideline-based dietary interventions. During this interactive roundtable discussion, expert faculty will discuss this challenge, how they address it, as well as how the treatment landscape for LC-FAODs is evolving to enable better outcomes for patients.
A Medscape LIVE symposium supported by an educational grant from Ultragenyx
Sponsored by
Sponsored Satellite Symposium 3:
One year of treatment experience with PALYNZIQ®▼ (pegvaliase) in Europe
Welcome
Ania C. Muntau (Hamburg, Germany)
One year of treatment experience with PALYNZIQ in Europe
Ania C. Muntau (Hamburg, Germany)
Live Q&A
Faculty & audience
This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. Healthcare professionals should report adverse events in accordance with their local requirements. Adverse events should also be reported to BioMarin on + 1 415 506 6179 or drugsafety@bmrn.com.
Sponsored by
12.45-13.15 GMT
Sponsored Satellite Symposium 4:
Re-defining management of alkaptonuria: Transforming the outlook for patients with the iconic Mendelian disease - a tribute to Archibald Garrod
DevelopAKUre: Disease-modifying pharmacological treatment of AKU with nitisinone – results from an international, randomised controlled Phase 3 study (SONIA 2)
Lakshminarayan Ranganath (Liverpool, United Kingdom)
AKU from the patients’ perspective – Impact of AKU symptoms as perceived by the patients
Nicolas Sireau (Cambridge, United Kingdom)
Sponsored by
Sponsored Satellite Symposium 5:
Shortening the Journey to Fabry Disease Diagnosis: Perspectives from a Patient and Nephrologist
David G. Warnock (Birmingham, United States)
Paul R.
Fabry Patient (United States)
Sponsored by
Sponsored Satellite Symposium 6:
Optimizing IEM Care During a Pandemic: A Case-Based Look at UCD Management
A case-based program that reviews key aspects of urea cycle disorders (UCD) and illustrates real-world challenges and strategies for successful management of inborn errors of metabolism during the pandemic.
Speaker:
Gregory Enns, MD (Stanford University, Lucile Salter Packard Children’s Hospital, Palo Alto, CA)
Sponsored by
20.00-21.00 GMT
Sponsored Satellite Symposium 7:
Distinguishing Pediatric Movement Disorders - Uncovering AADC Deficiency
Welcome and introductions
Sunay Ozdas
Distinguishing AADC deficiency: Clinical experience with differential diagnosis
Berrin Monteleone
Current management strategies and emerging therapies for AADC deficiency
Wuh-Liang Hwu
Q&A panel discussion
Sunay Ozdas
Berrin Monteleone
Symposium Objectives:
- Review pediatric disorders that may clinically resemble AADC deficiency
- Share clinical experience with differential diagnosis of AADC deficiency
- Discuss current management options and treatments in development for AADC deficiency
Sponsored by
Sponsored Satellite Symposium 8:
Fabry phenotypes: GLA variants in the real world
Welcome & introduction
Jörn Schenk (Zurich, Switzerland)
What can GLA variants tell us and what can’t they?
Gheona Altarescu (Jerusalem, Israel)
Working with phenotypic variability
Derralynn Hughes (London, United Kingdom)
Live Q&A
Sponsored by
20.00-20.30 GMT
Sponsored Satellite Symposium 9:
Long-Term Management of Organic Acidemia Patients With CARBAGLU®: The PROTECT Trial Overview & Interim Analysis.
Chair: Johannes Häberle (Zurich, Switzerland)
Introduction & Unmet needs in the long-term management of organic acidurias
Johannes Häberle (Zurich, Switzerland)
PROTECT Trial - Overview & enrolment status
Sufin Yap (Sheffield, United Kingdom)
PROTECT Trial - Interim analysis results
Sufin Yap (Sheffield, United Kingdom)
Open discussion & Q&A
Sponsored by
