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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

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors

K John Pasi, Pencho Georgiev, Tim Mant, Toshko Lissitchkov, Michael Desmond Creagh, David Bevan, Steve Austin, Charles R Hay, Inga Hegemann, Rashid Kazmi, Pratima Chowdary, Margaret V. Ragni, MD, Chang-Heok Soh, Akin Akinc, Angela M. Partisano, Benny Sorenson, Savita Rangarajan
Journal: Blood
Blood (2016) 128 (22): 1397.
https://doi.org/10.1182/blood.V128.22.1397.1397
Published: 2016
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

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients without Inhibitors

Margaret V. Ragni, MD, Pencho Georgiev, Tim Mant, Michael Desmond Creagh, Toshko Lissitchkov, David Bevan, Steve Austin, Charles R Hay, Inga Hegemann, Rashid Kazmi, Pratima Chowdary, Savita Rangarajan, Chang-Heok Soh, Akin Akinc, Angela M. Partisano, Benny Sorenson, K John Pasi
Journal: Blood
Blood (2016) 128 (22): 2572.
https://doi.org/10.1182/blood.V128.22.2572.2572
Published: 2016