The Voice of the Patient Report: PFDD Meeting
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The Voice of the Patient Report: PFDD Meeting
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In this week’s episode we look back at the PFDD meeting that led to the Voice of The Patient Report, a landmark document in the fight for better treatment options for those living with Classic Galactosemia
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Watch the full PFDD meeting or download the VOP Report here: https://rarediseases.org/externally-led-patient-focused-drug-development-meeting-for-galactosemia/
00:00 – Introduction
04:00 – Welcome from NORD and the Galactosemia Foundation
05:00 – A History of the Galactosemia Foundation
06:00 – Defining “Unmet Needs” for Classic Galactosemia
07:00 – Clinical Overview with Dr. Judy Fridovich-Keil
13:00 – The Impact of Dietary Management: Life-Saving but Not Enough
17:00 – Long-Term Complications Despite Early Intervention
20:00 – Dr. Sheila Farrah’s Remarks from the FDA
21:00 – First Panel: Family Stories Begin
22:00 – Jillian’s Story: Penelope’s Metabolic Crisis and Ongoing Trials
27:00 – Elaine’s Story: Loss, Disability, and Lifelong Care
28:00 – Cassidy’s Story: Slow Processing, Financial Strain
29:00 – Natalie’s Story: Two Children, Cerebral Palsy, and Hormone Therapy
30:00 – Maureen’s Story: POI and Lifelong Academic Struggles
31:00 – Community Discussion: Seizures, Delays, and Systemic Failures
32:00 – Amber’s Story: Clinical Trial Progress and Tremor Improvement
33:00 – Tiffany’s Story: Speech and Occupational Therapy Needs
34:00 – Heather’s Story: Treatment Access and Out-of-Pocket Costs
35:00 – Amy’s Story: Clinical Trial Experience and the Ethics of Placebo
36:00 – Final Reflections: The Need for Treatment Is Clear
37:00 – Brittany and Scott Close the Meeting: What Comes Next?