Garrod Symposium Agendas
Thursday, May 8, 2025
11:00–1:00
Registration Open
12:00
Lunch
12:20–12:25
Welcome to Symposium
Gabriella Horvath, Conference Chair
12:30–1:15
Lightening Talks
1:15–1:30
Break
Session 1: Understanding cellular models in preclinical stages of drug design, orphan drug development and precision therapy in rare disorders.
1:30–2:10
Precision therapy in rare disorders
Andreas Schulze or Damon Poburko
30-minute talk and 10 minutes Q & A
2:10–2:50
RNA lipid particle platform
Neel Mehta
30-minute talk and 10 minutes Q & A
2:50–3:30
Cellular models in precision therapy in IMD (iPSC)
Blair Levitt
30-minute talk and 10 minutes Q & A
3:30–4:00
Afternoon Break - Visit Posterboards
Session 2: Biomarker development for clinical trials, limitations and challenges
4:00–4:40
MS protein-based biomarkers
Mari Demarco
30-minute talk and 10 minutes Q & A
4:40–5:25
3 oral presentations selected from abstract submissions
5:25–6:00
Stable Isotope loading technique
Rajavel Elango
30-minute talk and 5 minutes Q & A
6:00–8:00
Welcome Reception and Poster Judging
7:15–9:15
Board of Directors Meeting (By invitation only)
Friday, May 9, 2025
7:30–8:30
Breakfast
8:30–9:30
Scriver Keynote Lectureship: Biomarkers for clinical trials in mitochondrial disorders
Shamima Rahman
50-minute talk and 10 minutes Q & A
Session 3: Drivers of change in newborn screening: novel therapies, new screening technologies, patient advocacy, new biomarker discovery
9:30–10:00
Efforts towards national coordination of newborn screening programs
Hilary Vallance
25-minute talk with 5 minutes Q & A
10:00–10:30
Biomarker and multiplex enzyme assay development for LSD newborn screening
Michael Gelb
25-minute talk with 5 minutes Q & A
10:30-11:00
Morning Break - Visit Posterboards
Session 4: TBC
11:00–11:45
Biomarker development for clinical trials in metabolic leukodystrophies Fanny Mochel (virtual)
30-minute talk and 10 minutes Q & A
11:45–12:30
3 oral presentations selected from abstract submissions
12:30–1:30
Lunch - Visit Posterboards
Session 5: Novel clinical trial designs, advances and failures (environmental sustainability)
1:30–2:10
Novel clinical trial designs
Beth Potter
30-minute talk and 10 minutes Q & A
2:10–2:50
Environmental sustainability in aspects of newborn screening and clinical trial design
Andre Mattman and Aneal Khan
30-minute talk and 10 minutes Q & A
2:50–3:30
Using Registry data and natural history studies, databases to have control group in clinical trials
Saadet Andrews
30-minute talk and 10 minutes Q & A
3:30–4:00
Afternoon Break - Visit Posterboards
Session 6: Artificial Intelligence in Precision Treatment in IMD
4:00–4:40
AI in precision treatment in IMD (overview)
Presenter - TBC
30-minute talk and 10 minutes Q & A
4:40–5:20
AI in clinical diagnostics of IMD
Wyeth Wasserman
30-minute talk and 10 minutes Q & A
5:20–6:00
AI in development of clinical severity scores as biomarkers for precision treatment
Presenter – TBC
30-minute talk and 10 minutes questions
7:00–11:00
Awards Dinner
Saturday, May 10, 2025
7:30–8:30
Breakfast
Session 7: Financial, Ethical, Legal, Social Issues
9:00–9:45
Directions for rare diseases clinical trials
(drug pricing negotiations, government policies, negotiations)
Speaker to TBC
9:45–10:30
Pillar 3 for CRDN (Care, support and empowerment – for patient advocacy supports)
Grant Mitchell
10:30–10:45
Morning Coffee Break - Visit Posterboards
10:45–12:00
Debate & Discussion: Surrogate endpoints as markers for drug efficacy
Presenters: Michael West & Shirin Rizzardo
Moderator: Sandra Sirrs
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12:00–12:30
Closing Remarks
Aziz Mhanni, President of Garrod Association
12:05–1:30
Garrod Association AGM with Lunch
All Members Welcome