50th ASH Annual Meeting and Exposition (December 6 - 9, 2008): Hemophilia: Clinical and Translational Insights

Monday, December 8, 2008: 1:30 PM-3:00 PM

3020-3022-3024 - West (Moscone Center)

Moderators:

Marilyn Manco-Johnson and Shawn M. Jobe

1:30 PM

Site-Specific PEGylation of Factor VIII (PEG-FVIII) Preserves Full Clotting Activity and Extends Therapeutic Efficacy in HemophiliaA Dogs

Tongyao Liu¹^*, David Lillicrap², Xin Zhang¹^*, Andrea Labelle²^*, Sandra Powell²^*, Baisong Mei¹^*, John E. Murphy¹^*, Glenn F. Pierce¹^* and Haiyan Jiang¹^*

¹Bayer HealthCare, Richmond, CA
²Richardson Laboratory, Queen's University, Kingston, ON, Canada

1:45 PM

512

Nonsense Suppression Approaches in Treating Hemophilia

Yi-Lin Liu¹^*, Paris Margaritis¹^*, Fayaz Khazi¹^*, Harre Downey¹^*, Stephan Kadauke¹^*, Nicole Hasbrouck¹^*, Josephine Sheedy²^*, Ellen Welch²^*, Marla Weetall²^* and Katherine A. High, MD³

¹Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA
²PTC Therapeutics Inc., South Plainfield, NJ
³Howard Hughes Medical Institute, The Children's Hospital of Philadelphia, Philadelphia, PA

2:00 PM

513

Most Factor VIII B Domain Missense Mutations Are Unlikely to Be Causative Mutations for Hemophilia A : Implications for Factor VIII Genetic Analysis

Kyoichi Ogata, MD¹^* and Steven W. Pipe, MD²

¹Pediatrics, University of Michigan, Ann Arbor, MI
²Pediatrics, L2110 Women's Hospital, Ann Arbor, MI

2:15 PM

514

Phenotypic Correction of Hemophilia a Using An Ips-Based Cellular Therapy

Dan Xu^*, Zaida Alipio^*, Jianchang Yang^*, Louis M. Fink, Wilson Xu^*, David C Ward^* and Yupo Ma

Nevada Cancer Institute, Las Vegas, NV

2:30 PM

515

Increased Thrombin Generation in Severe Hemophiliacs with Mild Clinical Phenotype

Elena Santagostino¹^*, Maria Elisa Mancuso¹^*, Armando Tripodi¹^*, Veena Chantarangkul¹^*, Gianluigi Pasta¹^*, Simona Maria Siboni¹^* and Pier M. Mannucci, MD²

¹Department of Medicine and Medical Specialties, IRCCS Maggiore Hospital, Mangiagalli & Regina Elena Foundation, Milan, Italy
²Department of Medicine and Medical Specialties, IRCCS Maggiore Hospital, Mangiagalli & Regina Elena Foundation, Milano, Italy

2:45 PM

516

Efficacy and Safety of Secondary Prophylactic Versus On-Demand Sucrose-Formulated Recombinant Factor VIII Treatment in Adults with Severe Hemophilia A: Results from a 13-Month Crossover Study

Peter Collins¹^*, Albert Faradji²^*, Massimo Morfini³^*, Monika Maas Enriquez, MD⁴^*, Eduard Gorina, MD⁵^*, Lawrence Schwartz⁶^* and Georg Lemm, MD, PhD⁵^*

¹University Hospital, Cardiff, United Kingdom
²Hautepierre Hospital, Strasbourg, France
³University Hospital, Firenze, Italy
⁴Bayer Healthcare AG, Wuppertal, Germany
⁵Bayer HealthCare Pharmaceuticals, Berkeley, CA
⁶Bayer HealthCare Pharmaceuticals, Montville, NJ

See more of: Oral and Poster Abstracts

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^*signifies non-member of ASH

322.O3.6 Hemophilia: Clinical and Translational Insights