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Journal Articles

Interim Data from a Randomized, Placebo Controlled, Phase 1 Study of Aln-AS1, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria

Eliane Sardh, MD PhD, Pauline Harper, MD PhD, Nabil Al-Tawil, MD, Manisha Balwani, MD MS, Karl Anderson, MD, Joseph Bloomer, MD, D. Montgomery Bissel, MD, Robert J. Desnick, MD, Charles J. Parker, MD, John D. Phillips, PhD, Herbert L. Bonkovsky, MD, Craig Penz, MA, Amy Chan, PhD, Chang-Heok Soh, PhD, William Querbes, PhD, Amy Simon, MD, Penelope Stein, MD PhD, David C. Rees, FRCP, FRCPath, MA, MBBS
Journal: Blood
Blood (2016) 128 (22): 2318.
https://doi.org/10.1182/blood.V128.22.2318.2318
Published: 2016
Journal Articles

High PR3 or ELA2 expression by CD34 + cells in advanced-phase chronic myeloid leukemia is associated with improved outcome following allogeneic stem cell transplantation and may improve PR1 peptide–driven graft-versus-leukemia effects

Agnes S. M. Yong, Katayoun Rezvani, Bipin N. Savani, Rhoda Eniafe, Stephan Mielke, John M. Goldman, A. John Barrett
Journal: Blood
Blood (2007) 110 (2): 770–775.
https://doi.org/10.1182/blood-2007-02-071738
Published: 2007
Journal Articles

Neonatal Hemolytic Anemia Due to Inherited Harderoporphyria: Clinical Characteristics and Molecular Basis

J. Lamoril, H. Puy, L. Gouya, R. Rosipal, V. Da Silva, B. Grandchamp, T. Foint, B. Bader-Meunier, J.P. Dommergues, J.C. Deybach, Y. Nordmann
Journal: Blood
Blood (1998) 91 (4): 1453–1457.
https://doi.org/10.1182/blood.V91.4.1453
Published: 1998