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Journal Articles

Rationale and Design of a Phase 3 Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

Rodney H Falk, MD, Morie A Gertz, MD, Merrill D Benson, MD, Gustavo Buchele, MD PhD, Michela Brambatti, MD MS, Sotirios Tsimikas, MD, Nicholas J Viney, Bsc, Li-Jung Tai, MD PhD, Cecilia Monteiro, MD PhD, Qingqing Yang, MS, Louis St L O'Dea, MB B.Ch. BAO, FRCP, Ewa Karwatowska-Prokopczuk, MD PhD, Eugene Schneider, MD, Richard S Geary, PhD, Brett P Monia, PhD

Blood (2019) 134 (Supplement_1): 5764.

https://doi.org/10.1182/blood-2019-129269

Published: 2019

Journal Articles

Hemophilia A Dogs Tolerant to Human Factor VIII Provide a Unique Model to Determine Efficacy and Safety of AAV Delivery of Novel Factor VIII Variants

Giang N. Nguyen, BS, Lauren E. Wimsey, DVM, Elizabeth P. Merricks, PhD, Katherine P. Ponder, MD, Timothy C. Nichols, MD, Denise E. Sabatino, PhD

Blood (2019) 134 (Supplement_1): 3628.

https://doi.org/10.1182/blood-2019-126085

Published: 2019

Journal Articles

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

Markus Weiller, Helen Wang, Sogué Coulibaly, Maria Schuster, Hanspeter Rottensteiner, Kefeng Sun, Marinee K Chuah, Thierry Vandendriessche, Friedrich Scheiflinger, Werner Höllriegl

Blood (2019) 134 (Supplement_1): 4633.

https://doi.org/10.1182/blood-2019-124734

Published: 2019

Journal Articles

A Proteomic Atlas of Cardiac Amyloidosis

Taxiarchis Kourelis, MD, Surendra Dasari, PhD, Ahmed U Fayyaz, MD, Martha Grogan, Marina Ramirez-Alvarado, Margaret Redfield, Omar F. abou Ezzeddine, MD, C.M., M.S., Angela Dispenzieri, MD, Ellen D. McPhail, MD

Blood (2019) 134 (Supplement_1): 1790.

https://doi.org/10.1182/blood-2019-124802

Published: 2019

Journal Articles

Generation of a Unique Cohort of Hemophilia A Dogs Tolerant to Human FVIII for Evaluating the Safety and Efficacy of AAV Delivery of Wild Type and Variant Human FVIII

Giang N. Nguyen, Lauren Wimsey, Elizabeth Merricks, Katherine P. Ponder, MD, Timothy C. Nichols, MD, Denise E. Sabatino, PhD

Blood (2018) 132 (Supplement 1): 2453.

https://doi.org/10.1182/blood-2018-99-113492

Published: 2018

Journal Articles

The Changing Face of Amyloidosis Referrals at a Tertiary Center over the Past 3 Decades

Andrew Staron, Lawreen H Connors, Frederick L Ruberg, John L. Berk, Lisa M Mendelson, Vaishali Sanchorawala, MD

Blood (2018) 132 (Supplement 1): 5536.

https://doi.org/10.1182/blood-2018-99-112193

Published: 2018

Journal Articles

Plasma Cell Disorders in Patients with Age-Related Transthyretin (ATTRwt) Amyloidosis

M Hasib Sidiqi, MBBS, Shaji K. Kumar, MD, Surendra Dasari, PhD, Ellen D. McPhail, MD, Francis K. Buadi, MB, CHB, Rahma Warsame, MD, Martha Q. Lacy, MD, David Dingli, MD PhD, Wilson I Gonsalves, MD, Prashant Kapoor, MD, Taxiarchis Kourelis, MD, Nelson Leung, MD, Eli Muchtar, MD, John A. Lust, MD PhD, Robert A. Kyle, MD, S. Vincent Rajkumar, MD, Morie A. Gertz, MD, Angela Dispenzieri, MD

Blood (2018) 132 (Supplement 1): 5610.

https://doi.org/10.1182/blood-2018-99-120058

Published: 2018

Journal Articles

Clinical Characteristics and Outcome of Patients with Wild-Type Transthyretin and AL Cardiac Amyloidosis Confirmed By Mass Spectrometry

Stephen Parkin, MD BSc, Michael A. Seidman, MD FRCPC, Margot Davis, MD FRCPC, Kevin Song, MD

Blood (2018) 132 (Supplement 1): 3126.

https://doi.org/10.1182/blood-2018-99-115317

Published: 2018

Journal Articles

Impact of Inotersen on Functioning and Activities of Daily Living for Patients with Hereditary TTR Amyloidosis: Results from a Double-Blind Placebo-Controlled Trial

Aaron Yarlas, PhD, Giampaolo Merlini, MD, Michelle K White, PhD, Asia Sikora Kessler, PhD, Andrew Lovley, MA, Spencer D Guthrie, MPH, MBA, Michael Pollock, Morie A. Gertz, MD

Blood (2018) 132 (Supplement 1): 4812.

https://doi.org/10.1182/blood-2018-99-119578

Published: 2018

Journal Articles

Long-Term Update from the Open-Label Extension of the NEURO-TTR Study in Patients with Hereditary Transthyretin Amyloidosis

Thomas Brannagan, Annabel K. Wang, Teresa Coelho, Marcia Waddington Cruz, Michael J. Polydefkis, Peter J. Dyck, Violaine Plante-Bordeneuve, John L. Berk, Fabio Barroso, Giampaolo Merlini, Isabel Conceição, Steven G. Hughes, Jesse G. Kwoh, Shiangtung W. Jung, Spencer Guthrie, Michael Pollock, Merrill D. Benson, Morie A. Gertz, MD

Blood (2018) 132 (Supplement 1): 498.

https://doi.org/10.1182/blood-2018-99-116995

Published: 2018

Journal Articles

Impact of Inotersen on Subgroups of Patients with Hereditary TTR Amyloidosis: Results from a Double-Blind Placebo-Controlled Trial

Aaron Yarlas, PhD, Giampaolo Merlini, MD, Michelle K White, PhD, Asia Sikora Kessler, PhD, Andrew Lovley, MA, Spencer D Guthrie, MPH, MBA, Michael Pollock, Morie A. Gertz, MD

Blood (2018) 132 (Supplement 1): 4803.

https://doi.org/10.1182/blood-2018-99-119216

Published: 2018

Journal Articles

99m Technetium-Hydroxy-Diphosphate Tracer (99mTcHDP) Bone Scintigraphy: An Easily Accessible, Rapid, Non-Invasive Diagnostic Tool for Cardiac Amyloidosis

Simon DJ Gibbs, MBBS, FRACP, FRCPA, Caroline Wong, MBBS, James Hare, MBBS FRACP, Hendrik Zimmet, MBBS FRACP, Jay Hocking, MBBS, FRACP, FRCPA, Anthony P Schwarer, MD MBBS (Hons), FRACP, FRCPA, Stephen B Ting, MB BS, FRACP, FRCPA, PhD

Blood (2016) 128 (22): 3557.

https://doi.org/10.1182/blood.V128.22.3557.3557

Published: 2016

Journal Articles

The C Domain Mediates the Export of FVIII from the Endoplasmic Reticulum By Interacting with MCFD2

Zhigang Liu, PhD, Chunlei Zheng, PhD, Denise E. Sabatino, PhD, Bin Zhang, PhD

Blood (2016) 128 (22): 255.

https://doi.org/10.1182/blood.V128.22.255.255

Published: 2016

Journal Articles

Concomitant Myeloproliferative Disorders (MPD) and Amyloidosis

Angeline J Pallante, MD, Nelson Leung, MD, Robert A. Kyle, MD, Ayalew Tefferi, MD, Stephen J. Russell, MD PhD, Thomas M. Habermann, MD, Martha Q. Lacy, MD, Suzanne R. Hayman, MD, Aref Al-Kali, MD, Craig Reeder, MD, Angela Dispenzieri, MD, Morie A. Gertz, MD, Francis K Buadi, MD, C. Christopher Hook, MD, David Dingli, MD PhD, Animesh Pardanani, MD PhD

Blood (2016) 128 (22): 5480.

https://doi.org/10.1182/blood.V128.22.5480.5480

Published: 2016

Journal Articles

Adeno-Associated Viral Vector Delivery of Optimized Human Factor VIII Achieves Therapeutic Factor VIII Levels in Non-Human Primates

Xavier M. Anguela, PhD, Liron Elkouby, PhD, Raffaella Toso, PhD, Marti DiPietro, MS, Robert J. Davidson, Katherine A. High, MD, Denise E. Sabatino, PhD

Blood (2015) 126 (23): 199.

https://doi.org/10.1182/blood.V126.23.199.199

Published: 2015

Journal Articles

Hyperactivatable Protein C Expression Partially Rescues the Embryonic Lethality of TFPI Null Mice but Surviving Mice Develop Severe Hydrocephalus

Susan A Maroney, DVM, PhD, Paul Ellery, PhD, Nicholas D. Martinez, BS, Mark Zogg, BS, Hartmut Weiler, Alan E. Mast, MD PhD

Blood (2015) 126 (23): 422.

https://doi.org/10.1182/blood.V126.23.422.422

Published: 2015

Journal Articles

Genetic Absence of Thrombin Receptor Par4 Overcomes the Obligate Requirement of EPCR in Mouse Placenta

Michelle M Storage, BS, Jianzhong An, MD, Helena Liang, Qiuhui Yang, MD PhD, Mark Zogg, BS, Min Zhan, MS, Hartmut Weiler, Rashmi Sood, PhD

Blood (2015) 126 (23): 425.

https://doi.org/10.1182/blood.V126.23.425.425

Published: 2015

Journal Articles

Diagnosis of Amyloidosis Subtype By Laser-Capture Microdissection (LCM) and Tandem Mass Spectrometry (MS/MS) Proteomic Analysis

Peter Mollee, MBBS, MMedSc, FRACP, FRCPA, Patricia Renaut, MBBS, FRCPA, Samuel Boros, MBBS, FRCPA, Dorothy Loo, MSc, Michelle Hill, PhD BSc (Hon), BA

Blood (2015) 126 (23): 1779.

https://doi.org/10.1182/blood.V126.23.1779.1779

Published: 2015

Journal Articles

Adeno-Associated Virus 8 (AAV8) Vector Genome Biodistribution into Body Fluids Following Single Intravenous Administration of BAX 335 Gene Therapy for Hemophilia B

Boyan Litchev, MD, Paul E. Monahan, MD, Anne Prener

Blood (2015) 126 (23): 2040.

https://doi.org/10.1182/blood.V126.23.2040.2040

Published: 2015

Journal Articles

Improved Factor VIII Expression with the Introduction of a PACE-Furin Variant into Codon-Optimized Human Factor VIII

Liron Elkouby, PhD, Robert J. Davidson, Paris Margaritis, DPhil, Katherine A. High, MD, Denise E. Sabatino, PhD

Blood (2014) 124 (21): 3498.

https://doi.org/10.1182/blood.V124.21.3498.3498

Published: 2014

NARROW

Rationale and Design of a Phase 3 Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

Hemophilia A Dogs Tolerant to Human Factor VIII Provide a Unique Model to Determine Efficacy and Safety of AAV Delivery of Novel Factor VIII Variants

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

A Proteomic Atlas of Cardiac Amyloidosis

Generation of a Unique Cohort of Hemophilia A Dogs Tolerant to Human FVIII for Evaluating the Safety and Efficacy of AAV Delivery of Wild Type and Variant Human FVIII

The Changing Face of Amyloidosis Referrals at a Tertiary Center over the Past 3 Decades

Plasma Cell Disorders in Patients with Age-Related Transthyretin (ATTRwt) Amyloidosis

Clinical Characteristics and Outcome of Patients with Wild-Type Transthyretin and AL Cardiac Amyloidosis Confirmed By Mass Spectrometry

Impact of Inotersen on Functioning and Activities of Daily Living for Patients with Hereditary TTR Amyloidosis: Results from a Double-Blind Placebo-Controlled Trial

Long-Term Update from the Open-Label Extension of the NEURO-TTR Study in Patients with Hereditary Transthyretin Amyloidosis

Impact of Inotersen on Subgroups of Patients with Hereditary TTR Amyloidosis: Results from a Double-Blind Placebo-Controlled Trial

99m Technetium-Hydroxy-Diphosphate Tracer (99mTcHDP) Bone Scintigraphy: An Easily Accessible, Rapid, Non-Invasive Diagnostic Tool for Cardiac Amyloidosis

The C Domain Mediates the Export of FVIII from the Endoplasmic Reticulum By Interacting with MCFD2

Concomitant Myeloproliferative Disorders (MPD) and Amyloidosis

Adeno-Associated Viral Vector Delivery of Optimized Human Factor VIII Achieves Therapeutic Factor VIII Levels in Non-Human Primates

Hyperactivatable Protein C Expression Partially Rescues the Embryonic Lethality of TFPI Null Mice but Surviving Mice Develop Severe Hydrocephalus

Genetic Absence of Thrombin Receptor Par4 Overcomes the Obligate Requirement of EPCR in Mouse Placenta

Diagnosis of Amyloidosis Subtype By Laser-Capture Microdissection (LCM) and Tandem Mass Spectrometry (MS/MS) Proteomic Analysis

Adeno-Associated Virus 8 (AAV8) Vector Genome Biodistribution into Body Fluids Following Single Intravenous Administration of BAX 335 Gene Therapy for Hemophilia B

Improved Factor VIII Expression with the Introduction of a PACE-Furin Variant into Codon-Optimized Human Factor VIII

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American Society of Hematology

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American Society of Hematology

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