Search Results - "Fradette, Stephanie"

Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS by Miller, Timothy, Cudkowicz, Merit, Shaw, Pamela J, Andersen, Peter M, Atassi, Nazem, Bucelli, Robert C, Genge, Angela, Glass, Jonathan, Ladha, Shafeeq, Ludolph, Albert L, Maragakis, Nicholas J, McDermott, Christopher J, Pestronk, Alan, Ravits, John, Salachas, François, Trudell, Randall, Van Damme, Philip, Zinman, Lorne, Bennett, C. Frank, Lane, Roger, Sandrock, Alfred, Runz, Heiko, Graham, Danielle, Houshyar, Hani, McCampbell, Alexander, Nestorov, Ivan, Chang, Ih, McNeill, Manjit, Fanning, Laura, Fradette, Stephanie, Ferguson, Toby A

“…In a phase 1–2 dose-escalation trial involving adults with ALS due to SOD1 mutations who received intrathecal tofersen (an antisense oligonucleotide) or…”
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Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study by De Vivo, Darryl C., Bertini, Enrico, Swoboda, Kathryn J., Hwu, Wuh-Liang, Crawford, Thomas O., Finkel, Richard S., Kirschner, Janbernd, Kuntz, Nancy L., Parsons, Julie A., Ryan, Monique M., Butterfield, Russell J., Topaloglu, Haluk, Ben-Omran, Tawfeg, Sansone, Valeria A., Jong, Yuh-Jyh, Shu, Francy, Staropoli, John F., Kerr, Douglas, Sandrock, Alfred W., Stebbins, Christopher, Petrillo, Marco, Braley, Gabriel, Johnson, Kristina, Foster, Richard, Gheuens, Sarah, Bhan, Ishir, Reyna, Sandra P., Fradette, Stephanie, Farwell, Wildon

“…•NURTURE is an ongoing study of nusinersen started in a presymptomatic stage of SMA.•All infants were ≥25 months old, and alive without permanent…”
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Neurofilament as a potential biomarker for spinal muscular atrophy by Darras, Basil T., Crawford, Thomas O., Finkel, Richard S., Mercuri, Eugenio, De Vivo, Darryl C., Oskoui, Maryam, Tizzano, Eduardo F., Ryan, Monique M., Muntoni, Francesco, Zhao, Guolin, Staropoli, John, McCampbell, Alexander, Petrillo, Marco, Stebbins, Christopher, Fradette, Stephanie, Farwell, Wildon, Sumner, Charlotte J.

“…Objective To evaluate plasma phosphorylated neurofilament heavy chain (pNF‐H) as a biomarker in spinal muscular atrophy (SMA). Methods Levels of pNF‐H were…”
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Nusinersen in Infants Who Initiate Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy (SMA): Interim Efficacy and Safety Results From the Phase 2 NURTURE Study (S25.001) by De Vivo, Darryl C., Topaloglu, Haluk, Swoboda, Kathryn J., Bertini, Enrico, Hwu, Wuh-Liang, Crawford, Thomas O., Foster, Richard, Bhan, Ishir, Fradette, Stephanie, Farwell, Wildon, Reyna, Sandra P.

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Annual bacterial community cycle in a seasonally ice-covered river reflects environmental and climatic conditions by Cruaud, Perrine, Vigneron, Adrien, Fradette, Marie-Stéphanie, Dorea, Caetano C., Culley, Alexander I., Rodriguez, Manuel J., Charette, Steve J.

“…Bacteria are key players in biogeochemical cycles and control water quality in freshwater ecosystems. Nevertheless, little is known about the identity and…”
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Working toward improved monitoring of Cryptosporidium and Giardia (oo)cysts in water samples: testing alternatives to elution and immunomagnetic separation from USEPA Method 1623.1 by Fradette, Marie-Stéphanie, Charette, Steve J

“…Abstract Objective This study was designed to find a method to enhance the recovery of Cryptosporidium spp. and Giardia spp. parasites from water samples for…”
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Annual Protist Community Dynamics in a Freshwater Ecosystem Undergoing Contrasted Climatic Conditions: The Saint-Charles River (Canada) by Cruaud, Perrine, Vigneron, Adrien, Fradette, Marie-Stéphanie, Dorea, Caetano C., Culley, Alexander I., Rodriguez, Manuel J., Charette, Steve J.

“…Protists are key stone components of aquatic ecosystems, sustaining primary productivity and aquatic food webs. However, their diversity, ecology and…”
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Detection of Cryptosporidium spp. and Giardia spp. in Environmental Water Samples: A Journey into the Past and New Perspectives by Fradette, Marie-Stéphanie, Culley, Alexander I., Charette, Steve J.

“…Among the major issues linked with producing safe water for consumption is the presence of the parasitic protozoa Cryptosporidium spp. and Giardia spp. Since…”
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Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS by Miller, Timothy M., Cudkowicz, Merit E., Genge, Angela, Shaw, Pamela J., Sobue, Gen, Bucelli, Robert C., Chiò, Adriano, Van Damme, Philip, Ludolph, Albert C., Glass, Jonathan D., Andrews, Jinsy A., Babu, Suma, Benatar, Michael, McDermott, Christopher J., Cochrane, Thos, Chary, Sowmya, Chew, Sheena, Zhu, Han, Wu, Fan, Nestorov, Ivan, Graham, Danielle, Sun, Peng, McNeill, Manjit, Fanning, Laura, Ferguson, Toby A., Fradette, Stephanie

“…The antisense molecule tofersen was tested intrathecally in 108 patients with ALS due to SOD1 mutations. In a subgroup with faster-progressing disease, there…”
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Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study by Benatar, Michael, Wuu, Joanne, Andersen, Peter M., Bucelli, Robert C., Andrews, Jinsy A., Otto, Markus, Farahany, Nita A., Harrington, Elizabeth A., Chen, Weiping, Mitchell, Adele A., Ferguson, Toby, Chew, Sheena, Gedney, Liz, Oakley, Sue, Heo, Jeong, Chary, Sowmya, Fanning, Laura, Graham, Danielle, Sun, Peng, Liu, Yingying, Wong, Janice, Fradette, Stephanie

“…Despite extensive research, amyotrophic lateral sclerosis (ALS) remains a progressive and invariably fatal neurodegenerative disease. Limited knowledge of the…”
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Amyotrophic Lateral Sclerosis (ALS) Genetic Testing: Current Landscape and Potential Needs (2691) by Salmon, Kristiana, Glass, Jonathan, Genge, Angela, Chockalingam, Priya, Runz, Heiko, Fradette, Stephanie, Cochrane, Thos, Kupelian, Varant

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Preventing amyotrophic lateral sclerosis: insights from pre-symptomatic neurodegenerative diseases by Benatar, Michael, Wuu, Joanne, McHutchison, Caroline, Postuma, Ronald B, Boeve, Bradley F, Petersen, Ronald, Ross, Christopher A, Rosen, Howard, Arias, Jalayne J, Fradette, Stephanie, McDermott, Michael P, Shefner, Jeremy, Stanislaw, Christine, Abrahams, Sharon, Cosentino, Stephanie, Andersen, Peter M, Finkel, Richard S, Granit, Volkan, Grignon, Anne-Laure, Rohrer, Jonathan D, McMillan, Corey T, Grossman, Murray, Al-Chalabi, Ammar, Turner, Martin R

“…Significant progress has been made in understanding the pre-symptomatic phase of amyotrophic lateral sclerosis. While much is still unknown, advances in other…”
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Tofersen in adults with SOD1-ALS: phase 3 VALOR trial and open-label extension results by Shaw, Pamela, Miller, Timothy, Cudkowicz, Merit, Genge, Angela, Sobue, Gen, Nestorov, Ivan, Graham, Dan- ielle, Fanning, Laura, Fradette, Stephanie, McNeill, Manjit

“…VALOR was a Phase 3, placebo-controlled trial to evaluate the clinical efficacy and safety of tofersen in adults with ALS and a confirmed SOD1 mutation…”
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Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study by van den Berg, Leonard H, Rothstein, Jeffrey D, Shaw, Pamela J, Babu, Suma, Benatar, Michael, Bucelli, Robert C, Genge, Angela, Glass, Jonathan D, Hardiman, Orla, Libri, Vincenzo, Mobach, Theodore, Oskarsson, Björn, Pattee, Gary L, Ravits, John, Shaw, Christopher E, Weber, Markus, Zinman, Lorne, Jafar-nejad, Paymaan, Rigo, Frank, Lin, Luan, Ferguson, Toby A, Gotter, Anthony L, Graham, Danielle, Monine, Michael, Inra, Jennifer, Sinks, Susie, Eraly, Satish, Garafalo, Steve, Fradette, Stephanie

“…Hexanucleotide repeat expansion of C9orf72 is a common genetic cause of amyotrophic lateral sclerosis (ALS). No C9orf72-targeted treatments are available…”
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Nusinersen in Infants Who Initiate Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy (SMA): Interim Results From the Phase 2 NURTURE Study (993) by Finkel, Richard S., De Vivo, Darryl C., Swoboda, Kathryn J., Bertini, Enrico, Hwu, Wuh-Liang, Foster, Richard, Bhan, Ishir, Fradette, Stephanie, Farwell, Wildon

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Design of a Phase 3, Randomized, Placebo-controlled Trial of Tofersen Initiated in Clinically Pre-symptomatic SOD1 Mutation Carriers with a Longitudinal Natural History Run-in (2285) by Benatar, Michael, Wuu, Joanne, Andersen, Peter M., Andrews, Jinsy, Bucelli, Robert C., Otto, Markus, Ferguson, Toby A., Chen, Weiping, Fanning, Laura, Graham, Danielle, Sun, Peng, Liu, Yingying, Wong, Janice, Fradette, Stephanie

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Tofersen, a SOD1 Antisense Oligonucleotide in Participants with ALS – Results From a Multiple Dose Study (657) by Cudkowicz, Merit, Miller, Timothy, Shaw, Pamela, Bennett, C. Frank, Lane, Roger, Chang, Ih, Liu, Yingying, Chen, Weiping, Nestorov, Ivan, Graham, Danielle, Fanning, Laura, McNeill, Manjit, Fradette, Stephanie, Ferguson, Toby

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The Miami Framework for ALS and related neurodegenerative disorders: an integrated view of phenotype and biology by Benatar, Michael, Wuu, Joanne, Huey, Edward D., McMillan, Corey T., Petersen, Ronald C., Postuma, Ronald, McHutchison, Caroline, Dratch, Laynie, Arias, Jalayne J., Crawley, Anita, Houlden, Henry, McDermott, Michael P., Cai, Xueya, Thakur, Neil, Boxer, Adam, Rosen, Howard, Boeve, Bradley F., Dacks, Penny, Cosentino, Stephanie, Abrahams, Sharon, Shneider, Neil, Lingor, Paul, Shefner, Jeremy, Andersen, Peter M., Al-Chalabi, Ammar, Turner, Martin R.

“…Increasing appreciation of the phenotypic and biological overlap between amyotrophic lateral sclerosis (ALS) and frontotemporal dementia, alongside evolving…”
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Publisher Correction: The Miami Framework for ALS and related neurodegenerative disorders: an integrated view of phenotype and biology by Benatar, Michael, Wuu, Joanne, Huey, Edward D., McMillan, Corey T., Petersen, Ronald C., Postuma, Ronald, McHutchison, Caroline, Dratch, Laynie, Arias, Jalayne J., Crawley, Anita, Houlden, Henry, McDermott, Michael P., Cai, Xueya, Thakur, Neil, Boxer, Adam, Rosen, Howard, Boeve, Bradley F., Dacks, Penny, Cosentino, Stephanie, Abrahams, Sharon, Shneider, Neil, Lingor, Paul, Shefner, Jeremy, Andersen, Peter M., Al-Chalabi, Ammar, Turner, Martin R.

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Association of Phosphorylated Neurofilament Heavy Chain (pNF-H) Levels With Motor Function Achievement in Individuals With Spinal Muscular Atrophy (SMA) Treated With Nusinersen (S27.009) by Sumner, Charlotte J., Darras, Basil T., Muntoni, Francesco, Crawford, Thomas O., Finkel, Richard S., Mercuri, Eugenio, De Vivo, Darryl C., Oskoui, Maryam, Tizzano, Eduardo, Ryan, Monique M., Zhao, Guolin, Petrillo, Marco, Stebbins, Christopher, Fradette, Stephanie, Farwell, Wildon

“…Abstract only…”
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