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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

12:00–12:30

Closing Remarks

Aziz Mhanni, President of Garrod Association

 

12:05–1:30

Garrod Association AGM with Lunch

All Members Welcome

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