6 results on '"Anne Katrine Andreasen"'
Search Results
2. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment
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Esther M. Maier, Rosalia M Da Riol, Nikolaj H.T. Petersen, Saikat Santra, Federica Deodato, Paul Harmatz, Anne Katrine Andreasen, Thomas Hansen, Matthias Gautschi, Thomas Blaettler, Thomas Kirkegaard, Simon Day, Mireia del Toro, Christine í Dali, Sabine Grønborg, Marie Aavang Geist, Linda Ingemann, Stephanie Grunewald, Agathe Roubertie, Marc C. Patterson, Bénédicte Héron, Eugen Mengel, Anna Tylki-Szymańska, Mayo Clinic [Rochester], Ospedale 'Santa Maria della Misericordia' = University Hospital 'Santa Maria della Misericordia', Vall d'Hebron University Hospital [Barcelona], IRCCS Ospedale Pediatrico Bambino Gesù [Roma], Bern University Hospital [Berne] (Inselspital), Institute of Child Health [London], University College of London [London] (UCL), Rigshospitalet [Copenhagen], Copenhagen University Hospital, UCSF Benioff Children's Hospital Oakland, University of California [San Francisco] (UCSF), University of California-University of California, Service de Neuropédiatrie [CHU Trousseau], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-CHU Trousseau [APHP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Sorbonne Université (SU), Children's Hospital [Munich, Allemagne], Helmholtz-Zentrum München (HZM)-Technische Universität München [München] (TUM), CHU Montpellier, Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier), Birmingham Children’s Hospital, Children’s Memorial Health Institute [Warsaw, Poland] (CMHI), and Helmholtz-Zentrum München (HZM)-Technische Universität Munchen - Université Technique de Munich [Munich, Allemagne] (TUM)
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Male ,Internationality ,[SDV]Life Sciences [q-bio] ,heat shock protein ,Arimoclomol ,Type C ,Severity of Illness Index ,NPC clinical severity scale ,double-blindplacebo-controlled ,chemistry.chemical_compound ,0302 clinical medicine ,Miglustat ,Clinical endpoint ,arimoclomol ,Prospective Studies ,Child ,Genetics (clinical) ,Genetics & Heredity ,0303 health sciences ,Niemann-Pick disease type C ,Niemann-Pick Disease, Type C ,3. Good health ,Treatment Outcome ,6.1 Pharmaceuticals ,Child, Preschool ,Disease Progression ,Biomarker (medicine) ,biomarker ,Female ,medicine.symptom ,medicine.drug ,medicine.medical_specialty ,Adolescent ,Clinical Trials and Supportive Activities ,Clinical Sciences ,610 Medicine & health ,Placebo ,Hydroxylamines ,03 medical and health sciences ,Young Adult ,Double-Blind Method ,Clinical Research ,Internal medicine ,Niemann-Pick Disease ,Genetics ,medicine ,Humans ,Adverse effect ,Preschool ,030304 developmental biology ,Angioedema ,business.industry ,Evaluation of treatments and therapeutic interventions ,Confidence interval ,chemistry ,business ,030217 neurology & neurosurgery - Abstract
Niemann-Pick disease type C (NPC) is a rare, genetic, progressive neurodegenerative disorder with high unmet medical need. We investigated the safety and efficacy of arimoclomol, which amplifies the heat shock response to target NPC protein misfolding and improve lysosomal function, in patients with NPC. In a 12-month, prospective, randomised, double-blind, placebo-controlled, phase 2/3 trial (ClinicalTrials.gov identifier: NCT02612129), patients (2-18 years) were randomised 2:1 to arimoclomol:placebo, stratified by miglustat use. Routine clinical care was maintained. Arimoclomol was administered orally three times daily. The primary endpoint was change in 5-domain NPC Clinical Severity Scale (NPCCSS) score from baseline to 12 months. Fifty patients enrolled; 42 completed. At month 12, the mean progression from baseline in the 5-domain NPCCSS was 0.76 with arimoclomol vs 2.15 with placebo. A statistically significant treatment difference in favour of arimoclomol of -1.40 (95% confidence interval: -2.76, -0.03; P=.046) was observed, corresponding to a 65% reduction in annual disease progression. In the prespecified subgroup of patients receiving miglustat as routine care, arimoclomol resulted in stabilisation of disease severity over 12 months with a treatment difference of -2.06 in favour of arimoclomol (P=.006). Adverse events occurred in 30/34 patients (88.2%) receiving arimoclomol and 12/16 (75.0%) receiving placebo. Fewer patients had serious adverse events with arimoclomol (5/34, 14.7%) vs placebo (5/16, 31.3%). Treatment-related serious adverse events (n=2) included urticaria and angioedema. Arimoclomol provided a significant and clinically meaningful treatment effect in NPC and was well tolerated.
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- 2021
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3. Persistent Effect of Arimoclomol in Patients with Niemann-Pick Disease Type C: 24-Month Results from an Open-Label Extension of a Pivotal Phase 2/3 Study
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Eugen Mengel, Anna Tylki-Szymańska, Marie Aavang Geist, Esther M. Maier, Bénédicte Héron, Thomas Kirkegaard, Matthias Gautschi, Anne Katrine Andreasen, Simon Day, Stephanie Grunewald, Nikolaj H.T. Petersen, Mireia del Toro, Christine í Dali, Federica Deodato, Thomas Hansen, Marc C. Patterson, Saikat Santra, Paul Harmatz, Thomas Blaettler, Rosalia Maria Da Riol, Sabine Grønborg, Linda Ingemann, and Agathe Roubertie
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chemistry.chemical_compound ,medicine.medical_specialty ,Niemann–Pick disease, type C ,chemistry ,business.industry ,Internal medicine ,medicine ,In patient ,Arimoclomol ,Open label ,medicine.disease ,business ,Gastroenterology - Published
- 2021
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4. Persistent effect of arimoclomol in patients with Niemann-Pick disease type C: 24-month results from an open-label extension of a pivotal phase 2/3 study
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Marc Patterson, Eugen Mengel, Rosalia M. Da Rio, Mireia Del Toro, Federica Deodato, Matthias Gautschi, Stephanie Grunewald, Sabine Grønborg, Paul Harmatz, Bénédicte Héron, Esther M. Maier, Agathe Roubertie, Saikat Santra, Anna Tylki-Szymańska, Anne Katrine Andreasen, Marie Aavang Geist, Nikolaj Havnsøe Torp Petersen, Linda Ingemann, Thomas Hansen, Thomas Blaettler, Thomas Kirkegaard, and Christine Í. Dali
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Endocrinology ,Endocrinology, Diabetes and Metabolism ,Genetics ,Molecular Biology ,Biochemistry - Published
- 2022
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5. Structural and cognitive correlates of fatigue in progressive multiple sclerosis
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Volkert Siersma, Pernille Iversen, Lisbet Marstrand, Anne Katrine Andreasen, Hartwig R. Siebner, and Finn Sellebjerg
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Adult ,Male ,0301 basic medicine ,medicine.medical_specialty ,Prefrontal Cortex ,Primary Progressive Multiple Sclerosis ,03 medical and health sciences ,0302 clinical medicine ,Physical medicine and rehabilitation ,Parietal Lobe ,Humans ,Medicine ,Cognitive Dysfunction ,Secondary progressive ,Fatigue ,Progressive multiple sclerosis ,medicine.diagnostic_test ,business.industry ,Multiple sclerosis ,Motor Cortex ,Magnetic resonance imaging ,Cognition ,General Medicine ,Middle Aged ,Multiple Sclerosis, Chronic Progressive ,medicine.disease ,Magnetic Resonance Imaging ,030104 developmental biology ,Neurology ,Female ,Neurology (clinical) ,business ,030217 neurology & neurosurgery - Abstract
Fatigue in multiple sclerosis (MS) is a debilitating symptom and experienced by most patients. In recent studies investigating this phenomenon, the majority of patients had a relapsing-remitting disease course.Patients with progressive MS participating in one of three treatment trials during a period from 2010 to 2014 were included. Fatigue was assessed with the Fatigue Scale for Motor and Cognitive Functions (FSMC) and patients were further examined with a cognitive test battery, including Symbol Digit Modalities Test (SDMT), and 3 T MRI with subsequent quantitative analyses of 13 cortical regions of interest, deep grey matter and lesion volume.Twenty-two patients were enrolled. The thickness of the pre-central gyrus correlated significantly with motor fatigue. We found only a non-significant trend towards a correlation between cognitive fatigue and the thickness of the pre-central gyrus, the parietal inferior supra-marginal gyrus and the opercular part of the inferior frontal gyrus. 36% of participants had impaired processing speed and 9% had normal function on all tests. The scores on the FSMC-cognitive scale were related to performance on SDMT.In this exploratory study of patients with progressive MS, fatigue was related to processing speed. Motor fatigue was also related to the cortical thickness of the primary motor cortex and there was a trend towards a relationship between cognitive fatigue and the thickness of cortical areas involved in attentional processes. Additional studies are needed to further elucidate the relationship between regional cortical atrophy, cognitive functioning and the perception of fatigue.FSMC: Motor and Cognitive Functions; MS: Multiple Sclerosis; SDMT: Symbol Digit Modalities Test; MRI: Magnetic Resonance Imaging; RRMS: Relapsing-Remitting Disease Course; EDSS: Kurtzke Expanded Disability Status Scale; FLAIR: Fluid Attenuated Inversion Recovery; NAWM: Normal-Appearing White Matter; CGM: Cortical Grey Matter; CTh: Cortical Thickness; ROIs: Regions of Interest; Raven: Raven Progressive Matrices; TM A: Trail Making A; TM B: Trail Making B; Rey: Rey Complex Figure; Similarities: WAIS III Similarities; Stroop: Stroop Colour Naming Test; BDI: Becks Depression Inventory II.
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- 2018
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6. Persistent effect of arimoclomol in patients with Nuemann-Pick disease type C: 12-month results from an open-label extension of a pivotal phase 2/3 study
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Anne Katrine Andreasen, Matthias Gautschi, Nikolaj H.T. Petersen, Federica Deodato, Bénédicte Héron, Thomas Hansen, Marc C. Patterson, Marie Aavang Geist, Thomas Kirkegaard, Stephanie Grunewald, Saikat Santra, Mireia del Toro, Eugen Mengel, Christine í Dali, Anna Tylki-Szymańska, Linda Ingemann, Agathe Roubertie, Rosalia Maria Da Riol, Simon Day, Paul Harmatz, Thomas Blaettler, Esther M. Maier, and Sabine Grønborg
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medicine.medical_specialty ,business.industry ,Endocrinology, Diabetes and Metabolism ,Phase (waves) ,Disease ,Extension (predicate logic) ,Arimoclomol ,Biochemistry ,Gastroenterology ,chemistry.chemical_compound ,Endocrinology ,chemistry ,Internal medicine ,Genetics ,medicine ,In patient ,Open label ,business ,Molecular Biology - Published
- 2021
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