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Alnylam Pharmaceuticals
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Transthyretin familial amyloid polyneuropathy (TTR-FAP) in Mallorca: a comparison between late- and early-onset disease

Journal Article published Dec 2016 in Journal of the Peripheral Nervous System volume 21 issue 4 on pages 352 to 356

Research funded by Alnylam Pharmaceuticals

Authors: Juan Buades-Reinés, Manuel Raya-Cruz, Cristina Gallego-Lezaún, Tomás Ripoll-Vera, Mercedes Usón-Martín, Hernán Andreu-Serra, Eugenia Cisneros-Barroso

RNAi-mediated reduction of hepatic Tmprss6 diminishes anemia and secondary iron overload in a splenectomized mouse model of β-thalassemia intermedia

Journal Article published 23 Mar 2018 in American Journal of Hematology volume 93 issue 6 on pages 745 to 750

Research funded by Alnylam Pharmaceuticals

Authors: Paul J. Schmidt, Kaifeng Liu, Gary Visner, Kevin Fitzgerald, Shannon Fishman, Tim Racie, Julia L. Hettinger, James S. Butler, Mark D. Fleming

Combination therapy with aTmprss6RNAi-therapeutic and the oral iron chelator deferiprone additively diminishes secondary iron overload in a mouse model of β-thalassemia intermedia

Journal Article published 23 Mar 2015 in American Journal of Hematology volume 90 issue 4 on pages 310 to 313

Research funded by Alnylam Pharmaceuticals

Authors: Paul J. Schmidt, Tim Racie, Mark Westerman, Kevin Fitzgerald, James S. Butler, Mark D. Fleming

Bleeding and liver transplantation outcomes in haemophilia

Journal Article published 4 Nov 2016 in Haemophilia volume 23 issue 2 on pages 230 to 237

Research funded by Baxalta | Bristol-Myers Squibb | Dimension | Sparks | CSL Behring | Alnylam Pharmaceuticals | Genentech Roche | Biogen | Vascular Medicine Institute | Novo Nordisk | Baxter International | Bayer

Authors: K. D. Mehta, M. V. Ragni

Family dynamics in transthyretin-related familial amyloid polyneuropathy Val30Met: Does genetic risk affect family functioning?

Journal Article published 14 Aug 2018 in Clinical Genetics volume 94 issue 5 on pages 401 to 408

Research funded by Alnylam Pharmaceuticals | Ionis Pharmaceuticals | Pfizer

Authors: Alice Lopes, Carla Rodrigues, Isabel Fonseca, Alexandra Sousa, Margarida Branco, Teresa Coelho, Jorge Sequeiros, Paula Freitas

Pilot randomized, non-inferiority, cross-over trial of once-weekly vs. three times-weekly recombinant factor VIII prophylaxis in adults with severe haemophilia A

Journal Article published 11 Dec 2016 in Haemophilia volume 23 issue 1 on pages e43 to e46

Research funded by Baxalta | Pfizer | BioMarin Pharmaceutical | Sparks | CSL Behring | Heart Blood and Lung Institute (NHLBI R34-HL105870) | National Institutes of Health | Alnylam Pharmaceuticals | Dimensions | Chugai Pharmaceutical | Shire | Grifols | Biogen | Genentech/Roche | Bethesda MD | Novo Nordisk | Octapharma | Baxter International | Bayer | Tacere Benitec

Authors: M. V. Ragni, J. G. Yabes, P. F. Fogarty, N. C. Josephson, C. M. Kessler, A. T. Neff, L. Raffini, K. Brummel-Ziedins, C. G. Moore

“Red-flag” symptom clusters in transthyretin familial amyloid polyneuropathy

Journal Article published Mar 2016 in Journal of the Peripheral Nervous System volume 21 issue 1 on pages 5 to 9

Research funded by Pfizer | Alnylam Pharmaceuticals | ISIS Pharmaceuticals

Authors: Isabel Conceição, Alejandra González-Duarte, Laura Obici, Hartmut H.-J. Schmidt, Damien Simoneau, Moh-Lim Ong, Leslie Amass

Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy

Journal Article published 11 Sep 2017 in BMC Neurology volume 17 issue 1

Research funded by Alnylam Pharmaceuticals

Authors: David Adams, Ole B. Suhr, Peter J. Dyck, William J. Litchy, Raina G. Leahy, Jihong Chen, Jared Gollob, Teresa Coelho

Assessing Modeled CO2 Retention and Rebreathing of a Facemask Designed for Efficient Delivery of Aerosols to Infants

Journal Article published 2012 in ISRN Pediatrics volume 2012 on pages 1 to 10

Research funded by Alnylam Pharmaceuticals

Authors: Christian Mundt, Alexander Sventitskiy, Jeffrey E. Cehelsky, Andrea B. Patters, Markus Tservistas, Michael C. Hahn, Gerd Juhl, John P. DeVincenzo

Dexamethasone, rituximab and cyclophosphamide for relapsed and/or refractory and treatment-naïve patients with Waldenstrom macroglobulinemia

Journal Article published 8 Aug 2017 in British Journal of Haematology volume 179 issue 1 on pages 98 to 105

Research funded by Pfizer | AbbVie | Acerta Pharma | Bristol-Myers Squibb | Novartis | Seattle Genetics | Sanofi | Millennium | Pharmacyclics, Inc | Onyx Pharmaceuticals | Prothena | Alnylam Pharmaceuticals | Spectrum Pharmaceuticals | Ionis Pharmaceuticals | Celldex | Janssen Pharmaceuticals | Celgene | Merck | Takeda Pharmaceuticals North America | Amgen | Karyopharm Therapeutics | Affimed

Authors: Jonas Paludo, Jithma P. Abeykoon, Shaji Kumar, Amanda Shreders, Sikander Ailawadhi, Morie A. Gertz, Taxiarchis Kourelis, Rebecca L. King, Craig B. Reeder, Nelson Leung, Robert A. Kyle, Francis K. Buadi, Thomas M. Habermann, David Dingli, Thomas E. Witzig, Angela Dispenzieri, Martha Q. Lacy, Ronald S. Go, Yi Lin, Wilson I. Gonsalves, Rahma Warsame, John A. Lust, S. Vincent Rajkumar, Stephen M. Ansell, Prashant Kapoor

Development of lipid nanoparticle formulations of siRNA for hepatocyte gene silencing following subcutaneous administration

Journal Article published Dec 2014 in Journal of Controlled Release volume 196 on pages 106 to 112

Research funded by Canadian Institutes of Health Research (CIHR FRN 111627) | Alnylam Pharmaceuticals

Authors: Sam Chen, Yuen Yi C. Tam, Paulo J.C. Lin, Alex K.K. Leung, Ying K. Tam, Pieter R. Cullis

5′-C-Malonyl RNA: Small Interfering RNAs Modified with 5′-Monophosphate Bioisostere Demonstrate Gene Silencing Activity

Journal Article published 13 Jan 2016 in ACS Chemical Biology volume 11 issue 4 on pages 953 to 960

Research funded by Alnylam Pharmaceuticals

Authors: Ivan Zlatev, Donald J. Foster, Jingxuan Liu, Klaus Charisse, Benjamin Brigham, Rubina G. Parmar, Vasant Jadhav, Martin A. Maier, Kallanthottathil G. Rajeev, Martin Egli, Muthiah Manoharan

Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA

Journal Article published Aug 2016 in Journal of Controlled Release volume 235 on pages 236 to 244

Research funded by Canadian Institutes of Health Research (CIHR FRN 111627) | Alnylam Pharmaceuticals

Authors: Sam Chen, Yuen Yi C. Tam, Paulo J.C. Lin, Molly M.H. Sung, Ying K. Tam, Pieter R. Cullis

Tissue remodeling after interference RNA mediated knockdown of transthyretin in a familial amyloidotic polyneuropathy mouse model

Journal Article published Nov 2016 in Neurobiology of Aging volume 47 on pages 91 to 101

Research funded by Alnylam Pharmaceuticals | Federación Española de Enfermedades Raras

Authors: Nádia Pereira Gonçalves, Paula Gonçalves, Joana Magalhães, Miguel Ventosa, Ana Varela Coelho, Maria João Saraiva

The Nonclinical Safety Profile of GalNAc-conjugated RNAi Therapeutics in Subacute Studies

Journal Article published 23 Aug 2018 in Toxicologic Pathology volume 46 issue 7 on pages 735 to 745

Research funded by Alnylam Pharmaceuticals

Authors: Maja M. Janas, Carole E. Harbison, Victoria K. Perry, Brenda Carito, Jessica E. Sutherland, Akshay K. Vaishnaw, Natalie D. Keirstead, Garvin Warner

Pharmacokinetics of a novel extended half-life glycoPEGylated factor IX, nonacog beta pegol (N9-GP) in previously treated patients with haemophilia B: results from two phase 3 clinical trials

Journal Article published 24 Feb 2017 in Haemophilia volume 23 issue 4 on pages 547 to 555

Research funded by Baxalta | Boehringer Ingelheim | Pfizer | LEO Pharma Research Foundation | Inspiration | CSL Behring | Alnylam Pharmaceuticals | Biogen Idec | Biotest | Chugai Pharmaceutical | Grifols | Roche | Swedish Orphan Biovitrum | Novo Nordisk | Octapharma | Bayer | SOBI

Authors: A. Tiede, F. Abdul-Karim, M. Carcao, P. Persson, W. H. O. Clausen, S. Kearney, T. Matsushita, C. Negrier, J. Oldenburg, E. Santagostino, G. Young

Does α-synuclein have a dual and opposing effect in preclinical vs. clinical Parkinson's disease?

Journal Article published Jun 2014 in Parkinsonism & Related Disorders volume 20 issue 6 on pages 584 to 589

Research funded by National Institutes of Health (2R01ES10751, R15NS043162-01A2) | Alnylam Pharmaceuticals | Medtronic, Inc. | Province of British Columbia | Life Labs | Genome British Columbia | National Parkinson Foundation

Authors: Katerina Markopoulou, Joanna M. Biernacka, Sebastian M. Armasu, Kari J. Anderson, J. Eric Ahlskog, Bruce A. Chase, Sun Ju Chung, Julie M. Cunningham, Matthew Farrer, Roberta Frigerio, Demetrius M. Maraganore

Structural basis for the synergy of 4′- and 2′-modifications on siRNA nuclease resistance, thermal stability and RNAi activity

Journal Article published 11 Aug 2018 in Nucleic Acids Research volume 46 issue 16 on pages 8090 to 8104

Research funded by Alnylam Pharmaceuticals | Vanderbilt University | National Institutes of Health (R01 GM074161)

Authors: Joel M Harp, Dale C Guenther, Anna Bisbe, Lydia Perkins, Shigeo Matsuda, Gopal R Bommineni, Ivan Zlatev, Donald J Foster, Nate Taneja, Klaus Charisse, Martin A Maier, Kallanthottathil G Rajeev, Muthiah Manoharan, Martin Egli

Divergent effects of glucose and fructose on hepatic lipogenesis and insulin signaling

Journal Article published 3 Oct 2017 in Journal of Clinical Investigation volume 127 issue 11 on pages 4059 to 4074

Research funded by National Instiute of Health (DK031036) | NIH (K12 HD000850) | Alnylam Pharmaceuticals (SRA)

Authors: Samir Softic, Manoj K. Gupta, Guo-Xiao Wang, Shiho Fujisaka, Brian T. O’Neill, Tata Nageswara Rao, Jennifer Willoughby, Carole Harbison, Kevin Fitzgerald, Olga Ilkayeva, Christopher B. Newgard, David E. Cohen, C. Ronald Kahn

Widespread suppression of huntingtin with convection-enhanced delivery of siRNA

Journal Article published Jan 2012 in Experimental Neurology volume 233 issue 1 on pages 463 to 471

Research funded by Alnylam Pharmaceuticals | Medtronic Neuromodulation

Authors: David K. Stiles, Zhiming Zhang, Pei Ge, Brian Nelson, Richard Grondin, Yi Ai, Peter Hardy, Peter T. Nelson, Andrei P. Guzaev, Mark T. Butt, Klaus Charisse, Verbena Kosovrasti, Lubomir Tchangov, Michael Meys, Martin Maier, Lubomir Nechev, Muthiah Manoharan, William F. Kaemmerer, Douglas Gwost, Gregory R. Stewart, Don M. Gash, Dinah W.Y. Sah