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Efficacy and Safety of Leriglitazone in Patients With Friedreich Ataxia: A Phase 2 Double-Blind, Randomized Controlled Trial (FRAMES).
Pandolfo M, Reetz K, Darling A, Rodriguez de Rivera FJ, Henry PG, Joers J, Lenglet C, Adanyeguh I, Deelchand D, Mochel F, Pousset F, Pascual S, Van den Eede D, Martin-Ugarte I, Vilà-Brau A, Mantilla A, Pascual M, Martinell M, Meya U, Durr A. Pandolfo M, et al. Neurol Genet. 2022 Nov 1;8(6):e200034. doi: 10.1212/NXG.0000000000200034. eCollection 2022 Dec. Neurol Genet. 2022. PMID: 36524101 Free PMC article.
TRIAL REGISTRATION INFORMATION: ClinicalTrials.gov: NCT03917225; EudraCT: 2018-004405-64; submitted April 17, 2019; first patient enrolled April 2, 2019. clinicaltrials.gov/ct2/show/NCT03917225?term=NCT03917225&draw=2&rank=1. CLASSIFICATION OF EVIDENCE: This study …
TRIAL REGISTRATION INFORMATION: ClinicalTrials.gov: NCT03917225; EudraCT: 2018-004405-64; submitted April 17, 2019; first patient enrolled A …
Central Nervous System Therapeutic Targets in Friedreich Ataxia.
Harding IH, Lynch DR, Koeppen AH, Pandolfo M. Harding IH, et al. Among authors: pandolfo m. Hum Gene Ther. 2020 Dec;31(23-24):1226-1236. doi: 10.1089/hum.2020.264. Hum Gene Ther. 2020. PMID: 33238751 Free PMC article. Review.
Understanding the temporal course of neuropathological changes is needed to identify areas of modifiable disease progression and the CNS and PNS locations that can be targeted at different time points. As most major targets of long-term therapy are in the CNS, this review …
Understanding the temporal course of neuropathological changes is needed to identify areas of modifiable disease progression and the CNS and …
Long-term safety and effectiveness of natalizumab treatment in clinical practice: 10 years of real-world data from the Tysabri Observational Program (TOP).
Butzkueven H, Kappos L, Wiendl H, Trojano M, Spelman T, Chang I, Kasliwal R, Jaitly S, Campbell N, Ho PR, Licata S; Tysabri Observational Program (TOP) Investigators. Butzkueven H, et al. J Neurol Neurosurg Psychiatry. 2020 Jun;91(6):660-668. doi: 10.1136/jnnp-2019-322326. Epub 2020 Mar 31. J Neurol Neurosurg Psychiatry. 2020. PMID: 32234967 Free PMC article.
OBJECTIVE: The Tysabri Observational Programme (TOP), which began >10 years ago, is an open-label, multinational, prospective observational study evaluating the long-term safety and effectiveness of natalizumab in relapsing-remitting multiple sclerosis patients. ...
OBJECTIVE: The Tysabri Observational Programme (TOP), which began >10 years ago, is an open-label, multinational, prospective observation …
Protocol of a randomized, double-blind, placebo-controlled, parallel-group, multicentre study of the efficacy and safety of nicotinamide in patients with Friedreich ataxia (NICOFA).
Reetz K, Hilgers RD, Isfort S, Dohmen M, Didszun C, Fedosov K, Kistermann J, Mariotti C, Durr A, Boesch S, Klopstock T, Rodríguez de Rivera Garrido FJ, Schöls L, Klockgether T, Pandolfo M, Korinthenberg R, Lavin P, Molenberghs G, Libri V, Giunti P, Festenstein R, Schulz JB; EFACTS or NICOFA study group. Reetz K, et al. Among authors: pandolfo m. Neurol Res Pract. 2019 Oct 15;1:33. doi: 10.1186/s42466-019-0038-9. eCollection 2019. Neurol Res Pract. 2019. PMID: 33324899 Free PMC article. Clinical Trial.
Nicotinamide acts as a histone deacetylase inhibitor. Dose escalation studies have shown, that short term treatment with dosages of up to 4 g/day increase the expression of FXN mRNA and frataxin protein up to the levels of asymptomatic heterozygous gene carriers. The long- …
Nicotinamide acts as a histone deacetylase inhibitor. Dose escalation studies have shown, that short term treatment with dosages of u …
Prediction of Survival With Long-Term Disease Progression in Most Common Spinocerebellar Ataxia.
Diallo A, Jacobi H, Cook A, Giunti P, Parkinson MH, Labrum R, Durr A, Brice A, Charles P, Marelli C, Mariotti C, Nanetti L, Panzeri M, Castaldo A, Rakowicz M, Rola R, Sulek A, Schmitz-Hübsch T, Schöls L, Hengel H, Baliko L, Melegh B, Filla A, Antenora A, Infante J, Berciano J, van de Warrenburg BP, Timmann D, Boesch S, Nachbauer W, Pandolfo M, Schulz JB, Bauer P, Jun-Suk K, Klockgether T, Tezenas du Montcel S. Diallo A, et al. Among authors: pandolfo m. Mov Disord. 2019 Aug;34(8):1220-1227. doi: 10.1002/mds.27739. Epub 2019 Jun 18. Mov Disord. 2019. PMID: 31211461
Long-term evolution of patient-reported outcome measures in spinocerebellar ataxias.
Jacobi H, du Montcel ST, Bauer P, Giunti P, Cook A, Labrum R, Parkinson MH, Durr A, Brice A, Charles P, Marelli C, Mariotti C, Nanetti L, Sarro L, Rakowicz M, Sulek A, Sobanska A, Schmitz-Hübsch T, Schöls L, Hengel H, Baliko L, Melegh B, Filla A, Antenora A, Infante J, Berciano J, van de Warrenburg BP, Timmann D, Szymanski S, Boesch S, Nachbauer W, Kang JS, Pandolfo M, Schulz JB, Melac AT, Diallo A, Klockgether T. Jacobi H, et al. Among authors: pandolfo m. J Neurol. 2018 Sep;265(9):2040-2051. doi: 10.1007/s00415-018-8954-0. Epub 2018 Jun 29. J Neurol. 2018. PMID: 29959555
INTRODUCTION: To study the long-term evolution of patient-reported outcome measures (PROMs) in the most common spinocerebellar ataxias (SCAs), we analyzed 8 years follow-up data of the EUROSCA Natural History Study, a cohort study of 526 patients with SCA1, SCA2, SCA3 and …
INTRODUCTION: To study the long-term evolution of patient-reported outcome measures (PROMs) in the most common spinocerebellar ataxia …
Effect of natalizumab on disease progression in secondary progressive multiple sclerosis (ASCEND): a phase 3, randomised, double-blind, placebo-controlled trial with an open-label extension.
Kapoor R, Ho PR, Campbell N, Chang I, Deykin A, Forrestal F, Lucas N, Yu B, Arnold DL, Freedman MS, Goldman MD, Hartung HP, Havrdová EK, Jeffery D, Miller A, Sellebjerg F, Cadavid D, Mikol D, Steiner D; ASCEND investigators. Kapoor R, et al. Lancet Neurol. 2018 May;17(5):405-415. doi: 10.1016/S1474-4422(18)30069-3. Epub 2018 Mar 12. Lancet Neurol. 2018. PMID: 29545067 Clinical Trial.
INTERPRETATION: Natalizumab treatment for secondary progressive multiple sclerosis did not reduce progression on the primary multicomponent disability endpoint in part 1, but it did reduce progression on its upper-limb component. Longer-term trials are needed to assess whe …
INTERPRETATION: Natalizumab treatment for secondary progressive multiple sclerosis did not reduce progression on the primary multicomponent …
A 22-Year Follow-up Study of Long-term Cardiac Outcome and Predictors of Survival in Friedreich Ataxia.
Pousset F, Legrand L, Monin ML, Ewenczyk C, Charles P, Komajda M, Brice A, Pandolfo M, Isnard R, Tezenas du Montcel S, Durr A. Pousset F, et al. Among authors: pandolfo m. JAMA Neurol. 2015 Nov;72(11):1334-41. doi: 10.1001/jamaneurol.2015.1855. JAMA Neurol. 2015. PMID: 26414159
Cardiac hypertrophy was present in 57.9% (77 of 133), and electrocardiography was normal in 6.8% (9 of 133). MAIN OUTCOMES AND MEASURES: Long-term cardiac outcome and predictors of survival in FRDA. RESULTS: After a mean (SD) follow-up of 10.5 (5.5) years (range, 0.6-23.0 …
Cardiac hypertrophy was present in 57.9% (77 of 133), and electrocardiography was normal in 6.8% (9 of 133). MAIN OUTCOMES AND MEASURES: Lon …
Long-term disease progression in spinocerebellar ataxia types 1, 2, 3, and 6: a longitudinal cohort study.
Jacobi H, du Montcel ST, Bauer P, Giunti P, Cook A, Labrum R, Parkinson MH, Durr A, Brice A, Charles P, Marelli C, Mariotti C, Nanetti L, Panzeri M, Rakowicz M, Sulek A, Sobanska A, Schmitz-Hübsch T, Schöls L, Hengel H, Baliko L, Melegh B, Filla A, Antenora A, Infante J, Berciano J, van de Warrenburg BP, Timmann D, Szymanski S, Boesch S, Kang JS, Pandolfo M, Schulz JB, Molho S, Diallo A, Klockgether T. Jacobi H, et al. Among authors: pandolfo m. Lancet Neurol. 2015 Nov;14(11):1101-8. doi: 10.1016/S1474-4422(15)00202-1. Epub 2015 Sep 13. Lancet Neurol. 2015. PMID: 26377379
As potential treatments for these diseases are being developed, precise knowledge of their natural history is needed. We aimed to study the long-term disease progression of the most common spinocerebellar ataxias: SCA1, SCA2, SCA3, and SCA6. ...
As potential treatments for these diseases are being developed, precise knowledge of their natural history is needed. We aimed to study the …
Characteristics and outcomes of patients with multiple cervical artery dissection.
Béjot Y, Aboa-Eboulé C, Debette S, Pezzini A, Tatlisumak T, Engelter S, Grond-Ginsbach C, Touzé E, Sessa M, Metso T, Metso A, Kloss M, Caso V, Dallongeville J, Lyrer P, Leys D, Giroud M, Pandolfo M, Abboud S; CADISP Group. Béjot Y, et al. Among authors: pandolfo m. Stroke. 2014 Jan;45(1):37-41. doi: 10.1161/STROKEAHA.113.001654. Epub 2013 Dec 10. Stroke. 2014. PMID: 24326451 Free article.
BACKGROUND AND PURPOSE: Little is known about factors contributing to multiple rather than single cervical artery dissections (CeAD) and their associated prognosis. METHODS: We compared the baseline characteristics and short-term outcome of patients with multiple to single …
BACKGROUND AND PURPOSE: Little is known about factors contributing to multiple rather than single cervical artery dissections (CeAD) and the …
12 results