Session IV: Clinical Trial Design Considerations
Panel IV-a Considerations for Clinical Trial Design Optimization in Alpha-1 Antitrypsin Deficiencyand Lysosomal Diseases
Moderators
: Gina Smith, Krista Casazza (C-Path)
Panelists
: Jon Hagstrom (Alpha-1 Foundation),Christine í Dali (Zevra), Gavin Imperato (FDA), Jonathan Jacoby (SOAR-NPC), Ed Marins (Takeda),Jeff Siegel (FDA), Yulia Yasinskaya (FDA)
This Critical Path Institute summit featured presentations and panel discussions with C-Path collaborators from industry, academia, global regulatory agencies, nurses, parents and patients. The workshop:
Explored the unmet needs and potential solutions relevant to specific aspects of type-1 diabetes, alpha-1 antitrypsin deficiency, lysosomal diseases, and neonatal indications.
Demonstrated the critical role data sharing plays in enhancing drug development for vulnerable patient populations with conditions that require interventions early in life.
Reviewed the current landscape of regenerative therapies, identifying regulatory opportunities that could expedite the development of such groundbreaking products.
Please check back in for recordings from our sessions, to be posted below.
Contact pediatricsadmin@c-path.org if you have any questions.
Негізгі бет SBS Day 2 - Session IV: Clinical Trial Design Considerations
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