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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2000 1
2003 1
2006 2
2007 1
2009 1
2012 2
2013 1
2014 1
2015 1
2016 3
2018 2
2019 2
2020 2
2021 1
2022 2
2023 2
2024 0

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20 results

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Page 1
Clinical advances of RNA therapeutics for treatment of neurological and neuromuscular diseases.
Holm A, Hansen SN, Klitgaard H, Kauppinen S. Holm A, et al. RNA Biol. 2022;19(1):594-608. doi: 10.1080/15476286.2022.2066334. Epub 2021 Dec 31. RNA Biol. 2022. PMID: 35482908 Free PMC article.
Here, we describe different strategies for delivering RNA drugs to the CNS and review recent advances in clinical development of ASO drugs and siRNA-based therapeutics for the treatment of neurological diseases and neuromuscular disorders.Abbreviations 2'-MOE: 2'-O-(2-meth …
Here, we describe different strategies for delivering RNA drugs to the CNS and review recent advances in clinical development of ASO …
Effects of curcumin on neurological diseases: focus on astrocytes.
Eghbaliferiz S, Farhadi F, Barreto GE, Majeed M, Sahebkar A. Eghbaliferiz S, et al. Pharmacol Rep. 2020 Aug;72(4):769-782. doi: 10.1007/s43440-020-00112-3. Epub 2020 May 27. Pharmacol Rep. 2020. PMID: 32458309 Review.
In light of this, our current review aims to discuss the role of astrocytes as essential players in neurodegenerative diseases and suggest that curcumin is capable of direct inhibition of astrocyte activity with a particular focus on its effects in Alexander disease …
In light of this, our current review aims to discuss the role of astrocytes as essential players in neurodegenerative diseases and suggest t …
Hypomyelination caused by a novel homozygous pathogenic variant in FOLR1: complete clinical and radiological recovery with oral folinic acid therapy and review of the literature.
Potic A, Perrier S, Radovic T, Gavrilovic S, Ostojic J, Tran LT, Thiffault I, Pastinen T, Schiffmann R, Bernard G. Potic A, et al. Orphanet J Rare Dis. 2023 Jul 13;18(1):187. doi: 10.1186/s13023-023-02802-6. Orphanet J Rare Dis. 2023. PMID: 37443037 Free PMC article. Review.
Here, we report the first two cases with genetically proven cerebral folate transport deficiency from South-Eastern Europe, describe the effect of oral folinic acid therapy on clinical and neuroradiological features and review the literature. RESULTS: Two siblings presente …
Here, we report the first two cases with genetically proven cerebral folate transport deficiency from South-Eastern Europe, describe the eff …
Astrocyte Dysfunction in Developmental Neurometabolic Diseases.
Olivera-Bravo S, Isasi E, Fernández A, Casanova G, Rosillo JC, Barbeito L. Olivera-Bravo S, et al. Adv Exp Med Biol. 2016;949:227-243. doi: 10.1007/978-3-319-40764-7_11. Adv Exp Med Biol. 2016. PMID: 27714692 Review.
Core protocol development for phase 2/3 clinical trials in the leukodystrophy vanishing white matter: a consensus statement by the VWM consortium and patient advocates.
Schoenmakers DH, Leferink PS, Vanderver A, Bonkowsky JL, Krägeloh-Mann I, Bernard G, Bertini E, Fatemi A, Fogel BL, Wolf NI, Skwirut D, Buck A, Holberg B, Saunier-Vivar EF, Rauner R, Dekker H, van Bokhoven P, Stellingwerff MD, Berkhof J, van der Knaap MS. Schoenmakers DH, et al. BMC Neurol. 2023 Aug 17;23(1):305. doi: 10.1186/s12883-023-03354-9. BMC Neurol. 2023. PMID: 37592248 Free PMC article.
Several investigational medicinal products are either in or about to enter clinical trial phase. Clinical trials in VWM pose serious challenges, as VWM has an episodic disease course; disease phenotype is highly heterogeneous and predictable only for early on …
Several investigational medicinal products are either in or about to enter clinical trial phase. Clinical trials in VWM …
Alexander disease with mild dorsal brainstem atrophy and infantile spasms.
Torisu H, Yoshikawa Y, Yamaguchi-Takada Y, Yano T, Sanefuji M, Ishizaki Y, Sawaishi Y, Hara T. Torisu H, et al. Brain Dev. 2013 May;35(5):441-4. doi: 10.1016/j.braindev.2012.06.009. Epub 2012 Jul 20. Brain Dev. 2013. PMID: 22818990
We present the case of a Japanese male infant with Alexander disease who developed infantile spasms at 8 months of age. ...Lamotrigine effectively controlled the infantile spasms and improved the abnormal EEG findings. Although most patients with infantile Alexan …
We present the case of a Japanese male infant with Alexander disease who developed infantile spasms at 8 months of age. ...Lam …
Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease.
Hagemann TL, Powers B, Mazur C, Kim A, Wheeler S, Hung G, Swayze E, Messing A. Hagemann TL, et al. Ann Neurol. 2018 Jan;83(1):27-39. doi: 10.1002/ana.25118. Epub 2018 Jan 14. Ann Neurol. 2018. PMID: 29226998 Free PMC article.
Here we investigate whether suppressing GFAP with antisense oligonucleotides could provide a therapeutic strategy for treating Alexander disease. METHODS: In this study, we use GFAP mutant mouse models of Alexander disease to test the efficacy of antis …
Here we investigate whether suppressing GFAP with antisense oligonucleotides could provide a therapeutic strategy for treating Alexander …
Identification of diffuse and focal brain lesions by clinical magnetic resonance spectroscopy.
Kingsley PB, Shah TC, Woldenberg R. Kingsley PB, et al. NMR Biomed. 2006 Jun;19(4):435-62. doi: 10.1002/nbm.1039. NMR Biomed. 2006. PMID: 16763970 Review.
The purpose of this paper is to facilitate the comparison of magnetic resonance (MR) spectra acquired from unknown brain lesions with published spectra in order to help identify unknown lesions in clinical settings. The paper includes lists of references for published MR s …
The purpose of this paper is to facilitate the comparison of magnetic resonance (MR) spectra acquired from unknown brain lesions with publis …
Symptomatic care of late-onset Alexander disease presenting with area postrema-like syndrome with prednisolone; a case report.
Zardadi S, Razmara E, Rasoulinezhad M, Babaei M, Ashrafi MR, Pak N, Garshasbi M, Tavasoli AR. Zardadi S, et al. BMC Pediatr. 2022 Jul 13;22(1):412. doi: 10.1186/s12887-022-03468-y. BMC Pediatr. 2022. PMID: 35831840 Free PMC article.
BACKGROUND: Alexander disease (AxD) is classified into AxD type I (infantile) and AxD type II (juvenile and adult form). ...Latter MRI at the age of 8 years showed enlarged area postrema lesion and bilateral middle cerebellar peduncles and dentate nuclei involvement …
BACKGROUND: Alexander disease (AxD) is classified into AxD type I (infantile) and AxD type II (juvenile and adult form). ...La …
20 results