A Phase 1B Trial in GBA1-Associated Parkinson's Disease of BIA-28-6156, a Glucocerebrosidase Activator.

Autor: den Heijer JM; Department of Neurology, Centre for Human Drug Research, Leiden, the Netherlands.; Leiden University Medical Centre, Leiden, the Netherlands., Kruithof AC; Department of Neurology, Centre for Human Drug Research, Leiden, the Netherlands.; Leiden University Medical Centre, Leiden, the Netherlands., Moerland M; Department of Neurology, Centre for Human Drug Research, Leiden, the Netherlands.; Leiden University Medical Centre, Leiden, the Netherlands., Walker M; Certara, QSP, Canterbury, United Kingdom., Dudgeon L; Lysosomal Therapeutics Inc., Cambridge, Massachusetts, USA., Justman C; Lysosomal Therapeutics Inc., Cambridge, Massachusetts, USA., Solomini I; Lysosomal Therapeutics Inc., Cambridge, Massachusetts, USA., Splitalny L; Lysosomal Therapeutics Inc., Cambridge, Massachusetts, USA., Leymarie N; Lysosomal Therapeutics Inc., Cambridge, Massachusetts, USA., Khatri K; Lysosomal Therapeutics Inc., Cambridge, Massachusetts, USA., Cullen VC; Lysosomal Therapeutics Inc., Cambridge, Massachusetts, USA., Hilt DC; Lysosomal Therapeutics Inc., Cambridge, Massachusetts, USA., Groeneveld GJ; Department of Neurology, Centre for Human Drug Research, Leiden, the Netherlands.; Leiden University Medical Centre, Leiden, the Netherlands., Lansbury P; Lysosomal Therapeutics Inc., Cambridge, Massachusetts, USA.; Department of Neurology, Harvard Medical School, Boston, Massachusetts, USA.

Publikováno v: Movement disorders : official journal of the Movement Disorder Society [Mov Disord] 2023 Jul; Vol. 38 (7), pp. 1197-1208. Date of Electronic Publication: 2023 May 17.

A randomized single and multiple ascending dose study in healthy volunteers of LTI-291, a centrally penetrant glucocerebrosidase activator.

Autor: den Heijer JM; Centre for Human Drug Research, Leiden, The Netherlands.; Leiden University Medical Centre, Leiden, The Netherlands., Kruithof AC; Centre for Human Drug Research, Leiden, The Netherlands.; Leiden University Medical Centre, Leiden, The Netherlands., van Amerongen G; Centre for Human Drug Research, Leiden, The Netherlands.; Leiden University Medical Centre, Leiden, The Netherlands., de Kam ML; Centre for Human Drug Research, Leiden, The Netherlands., Thijssen E; Centre for Human Drug Research, Leiden, The Netherlands.; Leiden University Medical Centre, Leiden, The Netherlands., Grievink HW; Centre for Human Drug Research, Leiden, The Netherlands., Moerland M; Centre for Human Drug Research, Leiden, The Netherlands.; Leiden University Medical Centre, Leiden, The Netherlands., Walker M; Certara, QSP, Canterbury, CT2 7FG, UK., Been K; Lysosomal Therapeutics Inc., Cambridge, MA, USA., Skerlj R; Lysosomal Therapeutics Inc., Cambridge, MA, USA., Justman C; Lysosomal Therapeutics Inc., Cambridge, MA, USA., Dudgeon L; Lysosomal Therapeutics Inc., Cambridge, MA, USA., Lansbury P; Lysosomal Therapeutics Inc., Cambridge, MA, USA.; Harvard Medical School, Boston, MA, USA., Cullen VC; Lysosomal Therapeutics Inc., Cambridge, MA, USA., Hilt DC; Lysosomal Therapeutics Inc., Cambridge, MA, USA., Groeneveld GJ; Centre for Human Drug Research, Leiden, The Netherlands.; Leiden University Medical Centre, Leiden, The Netherlands.

Publikováno v: British journal of clinical pharmacology [Br J Clin Pharmacol] 2021 Sep; Vol. 87 (9), pp. 3561-3573. Date of Electronic Publication: 2021 Mar 02.

A Large-Scale Full GBA1 Gene Screening in Parkinson's Disease in the Netherlands.

Autor: den Heijer JM; Centre for Human Drug Research, Leiden, The Netherlands.; Leiden University Medical Center, Leiden, The Netherlands., Cullen VC; Lysosomal Therapeutics Inc, Cambridge, Massachusetts, USA., Quadri M; Erasmus Medical Center, Rotterdam, The Netherlands.; Janssen Vaccines and Prevention, Leiden, The Netherlands., Schmitz A; GenomeScan B.V., Leiden, The Netherlands., Hilt DC; Lysosomal Therapeutics Inc, Cambridge, Massachusetts, USA., Lansbury P; Lysosomal Therapeutics Inc, Cambridge, Massachusetts, USA., Berendse HW; Amsterdam University Medical Centers, Amsterdam, The Netherlands., van de Berg WDJ; Amsterdam University Medical Centers, Amsterdam, The Netherlands., de Bie RMA; Amsterdam University Medical Centers, Amsterdam, The Netherlands., Boertien JM; University Medical Center Groningen, Groningen, The Netherlands., Boon AJW; Erasmus Medical Center, Rotterdam, The Netherlands., Contarino MF; Leiden University Medical Center, Leiden, The Netherlands.; Haga Teaching Hospital, The Hague, The Netherlands., van Hilten JJ; Leiden University Medical Center, Leiden, The Netherlands., Hoff JI; St. Antonius Ziekenhuis, Nieuwegein, The Netherlands., van Mierlo T; Spaarne Gasthuis, Haarlem, The Netherlands., Munts AG; Spaarne Gasthuis, Haarlem, The Netherlands., van der Plas AA; Alrijne Ziekenhuis, Leiden, The Netherlands., Ponsen MM; Meander Medical Center, Amersfoort, The Netherlands., Baas F; Leiden University Medical Center, Leiden, The Netherlands., Majoor-Krakauer D; Erasmus Medical Center, Rotterdam, The Netherlands., Bonifati V; Erasmus Medical Center, Rotterdam, The Netherlands., van Laar T; University Medical Center Groningen, Groningen, The Netherlands., Groeneveld GJ; Centre for Human Drug Research, Leiden, The Netherlands.; Leiden University Medical Center, Leiden, The Netherlands.

Publikováno v: Movement disorders : official journal of the Movement Disorder Society [Mov Disord] 2020 Sep; Vol. 35 (9), pp. 1667-1674. Date of Electronic Publication: 2020 Jul 02.

Anticholinergic delirium caused by topical homatropine ophthalmologic solution: confirmation by anticholinergic radioreceptor assay in two cases

Autor: Tune Le, Bylsma Fw, Hilt Dc

Publikováno v: The Journal of Neuropsychiatry and Clinical Neurosciences. 4:195-197

The authors report two patients with prolonged metabolic delirium presumably initiated by systemic absorption of homatropine from a topical ophthalmologic solution. Anticholinergic toxicity was confirmed by muscarinic radioreceptor assay in both case

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a426535e5658f7ed67f5051978243aa6
https://doi.org/10.1176/jnp.4.2.195

Study protocol of the GRoningen early-PD Ambroxol treatment (GREAT) trial: a randomized, double-blind, placebo-controlled, single center trial with ambroxol in Parkinson patients with a GBA mutation.

Autor: Siemeling, O.^1,2 (AUTHOR) o.siemeling@umcg.nl, Slingerland, S.^1,2 (AUTHOR), van der Zee, S.^1,2 (AUTHOR), van Laar, T.^1,2 (AUTHOR)

Publikováno v: BMC Neurology. 5/1/2024, Vol. 24 Issue 1, p1-10. 10p.