Search Results - "Gheuens, Sarah"

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy by Finkel, Richard S, Mercuri, Eugenio, Darras, Basil T, Connolly, Anne M, Kuntz, Nancy L, Kirschner, Janbernd, Chiriboga, Claudia A, Saito, Kayoko, Servais, Laurent, Tizzano, Eduardo, Topaloglu, Haluk, Tulinius, Már, Montes, Jacqueline, Glanzman, Allan M, Bishop, Kathie, Zhong, Z. John, Gheuens, Sarah, Bennett, C. Frank, Schneider, Eugene, Farwell, Wildon, De Vivo, Darryl C

“…In this phase 3 trial, among infants with spinal muscular atrophy, those who received nusinersen were more likely to achieve major motor milestones and less…”
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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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Safety and efficacy of natalizumab in patients with acute ischaemic stroke (ACTION): a randomised, placebo-controlled, double-blind phase 2 trial by Elkins, Jacob, Dr, Veltkamp, Roland, Prof, Montaner, Joan, Prof, Johnston, S Claiborne, Prof, Singhal, Aneesh B, Prof, Becker, Kyra, Prof, Lansberg, Maarten G, Prof, Tang, Weihua, PhD, Chang, Ih, PhD, Muralidharan, Kumar, MS, Gheuens, Sarah, MD, Mehta, Lahar, MD, Elkind, Mitchell S V, Prof

“…Summary Background In animal models of acute ischaemic stroke, blocking of the leukocyte-endothelium adhesion by antagonism of α4 integrin reduces infarct…”
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Role of CD4+ and CD8+ T-Cell Responses against JC Virus in the Outcome of Patients with Progressive Multifocal Leukoencephalopathy (PML) and PML with Immune Reconstitution Inflammatory Syndrome by GHEUENS, Sarah, BORD, Evelyn, KESARI, Santosh, SIMPSON, David M, GANDHI, Rajesh T, CLIFFORD, David B, BERGER, Joseph R, NGO, Long, KORALNIK, Igor J

“…Article Usage Stats Services JVI Citing Articles Google Scholar PubMed Related Content Social Bookmarking CiteULike Delicious Digg Facebook Google+ Mendeley…”
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Outcome and survival of asymptomatic PML in natalizumab‐treated MS patients by Dong‐Si, Tuan, Richman, Sandra, Wattjes, Mike P., Wenten, Made, Gheuens, Sarah, Philip, Jeffrey, Datta, Shoibal, McIninch, James, Bozic, Carmen, Bloomgren, Gary, Richert, Nancy

“…Objective As of 3 September 2013, 399 cases of natalizumab‐associated progressive multifocal leukoencephalopathy (PML) were confirmed in multiple sclerosis…”
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Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy by Finkel, Richard S., Mercuri, Eugenio, Darras, Basil T., Connolly, Anne M., Kuntz, Nancy L., Kirschner, Janbernd, Chiriboga, Claudia A., Saito, Kayoko, Servais, Laurent, Tizzano, Eduardo, Topaloglu, Haluk, Tulinius, Már, Montes, Jacqueline, Glanzman, Allan M., Bishop, Kathie, Zhong, Z. John, Gheuens, Sarah, Bennett, C. Frank, Schneider, Eugene, Farwell, Wildon, De Vivo, Darryl C.

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In Vivo Modulatory Action of Extracellular Glutamate on the Anticonvulsant Effects of Hippocampal Dopamine and Serotonin by Clinckers, Ralph, Gheuens, Sarah, Smolders, Ilse, Meurs, Alfred, Ebinger, Guy, Michotte, Yvette

“…Purpose: Our recent work (Clinckers et al., J Neurochem 2004;89:834–43) demonstrated that intrahippocampal perfusion of 2 nM dopamine or serotonin via a…”
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Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies by Darras, Basil T, Chiriboga, Claudia A, Iannaccone, Susan T, Swoboda, Kathryn J, Montes, Jacqueline, Mignon, Laurence, Xia, Shuting, Bennett, C Frank, Bishop, Kathie M, Shefner, Jeremy M, Green, Allison M, Sun, Peng, Bhan, Ishir, Gheuens, Sarah, Schneider, Eugene, Farwell, Wildon, De Vivo, Darryl C

“…OBJECTIVETo report results of intrathecal nusinersen in children with later-onset spinal muscular atrophy (SMA). METHODSAnalyses included children from a phase…”
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An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials by Darras, Basil T., Farrar, Michelle A., Mercuri, Eugenio, Finkel, Richard S., Foster, Richard, Hughes, Steven G., Bhan, Ishir, Farwell, Wildon, Gheuens, Sarah

“…Background Treatment with nusinersen has demonstrated significant and clinically meaningful benefits in clinical trials in infants and children with spinal…”
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Progressive multifocal leukoencephalopathy: why gray and white matter by Gheuens, Sarah, Wüthrich, Christian, Koralnik, Igor J

“…Since it was first described in 1958, progressive multifocal leukoencephalopathy (PML), a demyelinating disease of the brain caused by the polyomavirus JC…”
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Mitapivat versus Placebo for Pyruvate Kinase Deficiency by Al-Samkari, Hanny, Galactéros, Frédéric, Glenthøj, Andreas, Rothman, Jennifer A, Andres, Oliver, Grace, Rachael F, Morado-Arias, Marta, Layton, D. Mark, Onodera, Koichi, Verhovsek, Madeleine, Barcellini, Wilma, Chonat, Satheesh, Judge, Malia P, Zagadailov, Erin, Xu, Rengyi, Hawkins, Peter, Beynon, Vanessa, Gheuens, Sarah, van Beers, Eduard J

“…Pyruvate kinase deficiency, the most common genetic lesion in the glycolytic pathway, leads to chronic hemolytic anemia. Mitapivat, an oral agent, can activate…”
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Progressive multifocal leukoencephalopathy in individuals with minimal or occult immunosuppression by Gheuens, Sarah, Pierone, Gerald, Peeters, Patrick, Koralnik, Igor J

“…BackgroundProgressive multifocal leukoencephalopathy (PML) is a deadly demyelinating disease of the brain, caused by reactivation of the polyomavirus JC (JCV)…”
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Predictors of survival and functional outcomes in natalizumab-associated progressive multifocal leukoencephalopathy by Dong-Si, Tuan, Gheuens, Sarah, Gangadharan, Amy, Wenten, Made, Philip, Jeffrey, McIninch, James, Datta, Shoibal, Richert, Nancy, Bozic, Carmen, Bloomgren, Gary, Richman, Sandra, Weber, Thomas, Clifford, David B.

“…Natalizumab, a highly effective therapy for relapsing-remitting multiple sclerosis, is associated with a risk of progressive multifocal leukoencephalopathy…”
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Cross-Community Collaboration and Data Collection to Optimize Patient Care in Hemolytic Anemias by Andemariam, Biree, Badalamenti, Ninamaria, Blaylark, Rae, Colasanti, Ralph, Colombatti, Raffaella, Grace, Rachael F., Jonassaint, Charles R., Kuo, Kevin H.M., Levine, Laurice M, Lobo, Clarisse, Schyrver, Tamara, Sheth, Sujit, Trimnell, Cassandra, Watson, Alejandra, Welle, Jill, Woolford, Teonna, John, Holly, Davis, Janie, Zaidi, Ahmar U, Gallagher, Eva, Gheuens, Sarah, Shah, Nirmish

“…Background: Patient engagement is becoming increasingly important for all facets of healthcare, from drug development and approval, to ensuring equitable…”
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Effects of Dimethyl Fumarate on Brain Volume Change in Relapsing-remitting Multiple Sclerosis: a Pooled Analysis of the Phase 3 DEFINE and CONFIRM Studies (P3.2-064) by Nakamura, Kunio, Mokliatchouk, Oksana, Arnold, Douglas L., Yousry, Tarek A., Kappos, Ludwig, Richert, Nancy, Fisher, Elizabeth, Gheuens, Sarah

“…Abstract only…”
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RENEWED: Long-Term Electrophysiological and Clinical Outcomes in Participants Previously Enrolled in the Opicinumab Phase 2 Study RENEW (1599) by Aktas, Orhan, Ziemssen, Focke, Ziemssen, Tjalf, Comi, Giancarlo, Butzkueven, Helmut, Izquierdo, Guillermo, Klistorner, Alexander, Cheng, Wenting, Naylor, Maria L., Gheuens, Sarah, Zhu, Bing

“…Abstract only…”
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Long-Term Hemoglobin Response and Reduction in Transfusion Burden Are Maintained in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat by Grace, Rachael F., Glenthøj, Andreas, Barcellini, Wilma, Verhovsek, Madeleine, Rothman, Jennifer A., Morado, Marta, Layton, D. Mark, Andres, Oliver, Galactéros, Frédéric, van Beers, Eduard J., Onodera, Koichi, Viprakasit, Vip, Chonat, Satheesh, Judge, Malia P., Kosinski, Penelope A., Hawkins, Peter, Gheuens, Sarah, Xu, Emily, McGee, Bryan, Beynon, Vanessa, Al-Samkari, Hanny

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Nusinersen in Infants Diagnosed with Spinal Muscular Atrophy (SMA): Study Design and Initial Interim Efficacy and Safety Findings from the Phase 3 International ENDEAR Study (CCI.002) by Kuntz, Nancy, Farwell, Wildon, Zhong, Zhenshao John, Sun, Peng, Gheuens, Sarah, Schneider, Eugene, Finkel, Richard

“…Abstract only…”
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Early-Onset Osteopenia and Osteoporosis in Patients with Pyruvate Kinase Deficiency by Al-Samkari, Hanny, Grace, Rachael F., Glenthoej, Andreas, Andres, Oliver, Barcellini, Wilma, Galactéros, Frédéric, Kuo, Kevin H.M., Layton, D. Mark, Morado, Marta, Viprakasit, Vip, Dong, Yan, Tai, Feng, Hawkins, Peter, Gheuens, Sarah, Bowden, Chris, Porter, John B., van Beers, Eduard

“…Background: Hereditary pyruvate kinase (PK) deficiency results in lifelong hemolytic anemia and several significant comorbidities, the epidemiology of which…”
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Bone Mineral Density Remains Stable in Pyruvate Kinase Deficiency Patients Receiving Long-Term Treatment with Mitapivat by Al-Samkari, Hanny, Grace, Rachael F., Glenthoej, Andreas, Andres, Oliver, Barcellini, Wilma, Galactéros, Frédéric, Kuo, Kevin H.M., Layton, D. Mark, Morado, Marta, Viprakasit, Vip, Dong, Yan, Tai, Feng, Grekas, Liz, Khoja, Karishma, Gheuens, Sarah, McGee, Bryan, Porter, John B., Van Beers, Eduard J.

“…Background: Hereditary pyruvate kinase (PK) deficiency results in chronic hemolysis that may lead to reduced bone mineral density (BMD). Mitapivat (AG-348) is…”
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