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Amy Simon, MD
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Journal Articles

Eighteen-Month Interim Analysis of Efficacy and Safety of Givosiran, an RNAi Therapeutic for Acute Hepatic Porphyria, in the Envision Open Label Extension

David J. Kuter, MD, Sioban Keel, MD, Charles J. Parker, MD, David C. Rees, MD, Ulrich Stölzel, MD, Paolo Ventura, MD, Manisha Balwani, Laurent Gouya, MD, PhD, Amy Simon, MD, Shangbin Liu, PhD, John J Ko, PharmD,MSc, Sean Rhyee, MD, M.P.H, Samuel M. Silver, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 13.
https://doi.org/10.1182/blood-2020-134564
Published: 2020
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

A Subcutaneously Administered RNAi Therapeutic (ALN-AT3) Targeting Antithrombin for Treatment of Hemophilia: Interim Phase 1 Study Results in Healthy Volunteers and Patients with Hemophilia a or B

Benny Sorensen, MD PhD, Tim Mant, MD, Akin Akinc, PhD, Amy Simon, MD, Lauren Melton, Christian Lynam, Andrew Strahs, PhD, Alfica Sehgal, PhD, Renta Hutabarat, PhD, Prasoon Chaturvedi, PhD, Scott Barros, PhD, Akshay Vaishnaw, MD PhD, Aln-AT Investigators
Journal: Blood
Blood (2014) 124 (21): 693.
https://doi.org/10.1182/blood.V124.21.693.693
Published: 2014