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Journal Articles

Explore: A Prospective, Multinational Natural History Study of Patients with Acute Hepatic Porphyrias (AHP) with Recurrent Attacks

Eliane Sardh, MD PhD, Laurent Gouya, MD, Joseph Bloomer, MD, Manisha Balwani, MD MS, Dwight Bissell, MD, David C Rees, MA, MBBS, FRCP, Ulrich Stolzel, MD, John D. Phillips, PhD, Raili Kauppinen, MD PhD, Janneke Langendonk, MD PhD, Robert J. Desnick, MD PhD, Jean-Charles Deybach, MD, Herbert L. Bonkovsky, MD, Charles J. Parker, MD, H Naik, MS, CGC, Michael Badminton, MB, ChB, PhD, Penny Stein, MD PhD, Elizabeth Minder, MD, Jerzy Windyga, Pavel Martasek, MD PhD, Maria Domenica Cappellini, MD, Paolo Ventura, MD, Pauline Harper, MD PhD, Sverre Sandberg, MD PhD, Aasne Aarsand, PhD, Felix Alegre, MD, Aneta Ivanova, MD PhD, Neila Talbi, MD, Stephanie Rock, PhD, Amy Chan, PhD, Chang Heok Soh, PhD, Katherine A. McCarthy, W Querbes, PhD, Craig Penz, MA, Amy Simon, MD, Karl Anderson, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 2211.
https://doi.org/10.1182/blood.V130.Suppl_1.2211.2211
Published: 2017
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

Phase 1-2 Clinical Trial of a Recombinant AAV5 Vector Containing the Human FIX Gene in Patients with Severe or Moderately Severe Haemophilia B

Wolfgang A. Miesbach, MD PhD, Christian Meyer, MD PhD, Bart Nijmeijer, PhD, Florence Salmon, PhD, Nadina Grosios, PhD, Harald Petry, PhD, Frank W.G. Leebeek, MD PhD
Journal: Blood
Blood (2014) 124 (21): 5948.
https://doi.org/10.1182/blood.V124.21.5948.5948
Published: 2014
Journal Articles

A Pilot Study Investigating the Safety and Efficacy of Injectable Hemin (Panhematin®) in Patients with Low to Intermediate-1 Risk Myelodysplastic Syndrome.

Jamile Shammo
Journal: Blood
Blood (2009) 114 (22): 4859.
https://doi.org/10.1182/blood.V114.22.4859.4859
Published: 2009
Journal Articles

Identification and Characterization of Feline Acute Intermittent Porphyria: The First Naturally-Occurring Animal Model.

Sonia Clavero, PhD, David F Bishop, PhD, Mark E Haskins, VMD, PhD, Raili Kauppinen, MD, PhD, Urs Giger, PD, DMV, Robert J Desnick, MD
Journal: Blood
Blood (2009) 114 (22): 3014.
https://doi.org/10.1182/blood.V114.22.3014.3014
Published: 2009
Journal Articles

Identification of Three Novel Substitutions in the Erythroid Promoter of the HMBS Gene: A New Erythroid Variant of Human Acute Intermittent Porphyria?

Elena Di Pierro, PhD, Valeria Besana, Dr, Valentina Brancaleoni, Dr, Dario Tavazzi, Dr, Maria Domenica Cappellini, MD
Journal: Blood
Blood (2008) 112 (11): 4574.
https://doi.org/10.1182/blood.V112.11.4574.4574
Published: 2008
Journal Articles

Deferasirox Efficacy and Safety for the Treatment of Transfusion- Dependent Iron Overload in Patients with a Range of Rare Anemias.

John B Porter, Kai-Hsin Lin, Dany Habr, Gabor Domokos, Abdel Hmissi, Swee Lay Thein
Journal: Blood
Blood (2008) 112 (11): 1419.
https://doi.org/10.1182/blood.V112.11.1419.1419
Published: 2008
Journal Articles

Interval Hemin Therapy: Efficacy in Reducing Hospitalizations for Acute Attacks of Acute Intermittent Porphyria.

Rene Rothstein-Rubin, MD
Journal: Blood
Blood (2005) 106 (11): 3771.
https://doi.org/10.1182/blood.V106.11.3771.3771
Published: 2005
Journal Articles

Large Deletion of Chromosome 11 Causes Acute Intermittent Porphyria in an Italian Family.

Elena Di Pierro, PhD, Valeria Besana, BD, Valeria Moriondo, BD, Dario Tavazzi, PhD, Giovanna Casalgrande, PhD, Emilio Rocchi, MD, Maria D. Cappellini, MD
Journal: Blood
Blood (2005) 106 (11): 3543.
https://doi.org/10.1182/blood.V106.11.3543.3543
Published: 2005
Journal Articles

Hemin Prophylaxis for Patients with Frequently Recurring Acute Porphyric Attacks: Experience with Five Cases.

Harry S. Griffith, MD
Journal: Blood
Blood (2005) 106 (11): 3545.
https://doi.org/10.1182/blood.V106.11.3545.3545
Published: 2005
Journal Articles

A Case Report of Prophylactic Hemin Treatments in the Management of Patients with Life-Threatening Attacks of Acute Intermittent Porphyria.

Richard E. Mills, MD
Journal: Blood
Blood (2005) 106 (11): 3757.
https://doi.org/10.1182/blood.V106.11.3757.3757
Published: 2005
Journal Articles

Acute Intermittent Porphyria during Pregnancy: A Case Outlining the Safety of Administering Hematin Infusions.

Raymond J. Jensen, DO
Journal: Blood
Blood (2005) 106 (11): 3759.
https://doi.org/10.1182/blood.V106.11.3759.3759
Published: 2005
Journal Articles

Synergistic Up-Regulation of Heme Oxygenase-1 in Human Liver by Heme and siRNA to Bach1:Possible Therapeutic Implications.

Tahereh Ghaziani, MD, Ying Shan, MD, MPH, Richard W. Lambrecht, PhD, Herbert L. Bonkovsky, MD
Journal: Blood
Blood (2004) 104 (11): 1633.
https://doi.org/10.1182/blood.V104.11.1633.1633
Published: 2004
Journal Articles

Hematin administration to an adult with lead intoxication

JM Lamon, BC Frykholm, DP Tschudy
Journal: Blood
Blood (1979) 53 (5): 1007–1011.
https://doi.org/10.1182/blood.V53.5.1007.1007
Published: 1979