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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

Highly heterogeneous nature of δ-aminolevulinate dehydratase (ALAD) deficiencies in ALAD porphyria

Clinical Trials & Observations
Motoyoshi Maruno, Kazumichi Furuyama, Reiko Akagi, Yutaka Horie, Kuniaki Meguro, Luba Garbaczewski, Nicholas Chiorazzi, Manfred O. Doss, Alexandre Hassoun, Rudolf Mercelis, Luc Verstraeten, Pauline Harper, Ylva Floderus, Stig Thunell, Shigeru Sassa
Journal: Blood
Blood (2001) 97 (10): 2972–2978.
https://doi.org/10.1182/blood.V97.10.2972
Published: 2001