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Thursday, May 29th
9:30 AM - 10:30 AM East Coast USA Time
Exploring Expanded Access: Balancing Patient Needs and Regulatory Considerations
Location: Woodrow Wilson
Chair: Mariam Ahmed, PharmD, PhD (she/her/hers) – Takeda
Speaker: Mira (Mitze) Klingenberg, RN (she/her/hers) – Voices for ALS
Speaker: Mwango Kashoki, MD, MPH – Parexel International
Speaker: Indranil Bhattacharya, PhD (he/him/his) – Takeda
Rare Diseases (RD)Oncology (ONC)
Thursday, May 29th
9:30 AM - 10:30 AM East Coast USA Time
Challenges and Opportunities of RNA Therapeutics, A Roller Coaster from Discovery to Recovery I: Pioneering Patient-Centric RNA Therapeutics for Rare Diseases
Location: National Harbor 11
Chair: Hazem Hassan, PhD, RPh – Regeneron
Chair: Islam Younis, PhD – Merck & Co.
Speaker: Manoj Khurana, PhD – Alnylam Pharmaceuticals
Speaker: Chanchala Kaddi, PhD (she/her/hers) – Sanofi
Speaker: Hobart Rogers, PharmD, PhD – US Food and Drug Administration
Rare Diseases (RD)Cell, Gene, Regenerative Medicine & Nucleic Acid (CGRN)
Thursday, May 29th
3:30 PM - 4:30 PM East Coast USA Time
Balancing Patient-Centric and Model-Informed Drug Development (MIDD) Approaches to Optimize Dose Selection in Rare Diseases: Are We There Yet?
Location: Woodrow Wilson
Chair: Sathej Gopalakrishnan, PhD (he/him/his) – Merck KGaA
Chair: Noha Rayad, PhD – Alexion, AstraZeneca Rare Disease
Speaker: Roberta Smith, CNMT – Alagille Syndrome Alliance
Speaker: Rajesh Krishna, PhD, MBA – Certara
Speaker: Robert Schuck, PharmD, PhD – US Food and Drug Administration
Rare Diseases (RD)Pharmacometrics & Pharmacokinetics (PMK)
Friday, May 30th
9:30 AM - 10:30 AM East Coast USA Time
State of the Art Lecture - Michelle Werner
Location: Woodrow Wilson
State of the Art Speaker: Michelle Werner (she/her/hers) – Alltrna
Moderator: Sandra A.G Visser, PhD (she/her/hers) – Alltrna
Rare Diseases (RD)Cell, Gene, Regenerative Medicine & Nucleic Acid (CGRN)
Friday, May 30th
3:00 PM - 4:00 PM East Coast USA Time
Advancing Decentralized Clinical Trials for Rare Diseases and Special Populations: Perspectives and Opportunities
Location: National Harbor 10
Chair: Jackson K. Burton, PhD (he/him/his) – Biogen
Chair: Katty Wan, PhD (she/her/hers) – Pfizer
Speaker: Michelle Lorenz (she/her/hers) – Voices for ALS
Speaker: Laura Shaughnessy, PhD – UCB Biosciences, Inc
Speaker: Elimika Fletcher, PharmD, PhD – US Food and Drug Administration
Specific Populations (SPO)Rare Diseases (RD)