[HTML][HTML] The Alzheimer's disease drug development landscape

…, L Vermunt, PS Leferink, S Heetveld… - Alzheimer's Research & …, 2021 - Springer
Background Alzheimer’s disease (AD) is a devastating neurodegenerative disease leading
to dementia. The field has made significant progress over the last 15 years. AD diagnosis …

[HTML][HTML] Patterning factors during neural progenitor induction determine regional identity and differentiation potential in vitro

AG Nadadhur, PS Leferink, D Holmes, L Hinz… - Stem cell …, 2018 - Elsevier
The neural tube consists of neural progenitors (NPs) that acquire different characteristics
during gestation due to patterning factors. However, the influence of such patterning factors on …

Therapy trial design in vanishing white matter: an expert consortium opinion

…, P van Bokhoven, P van de Ven, PS Leferink - Neurology …, 2022 - AAN Enterprises
Vanishing white matter (VWM) is a leukodystrophy caused by recessive variants in the genes
EIF2B1-EIF2B5. It is characterized by chronic neurologic deterioration with superimposed …

Astrocyte subtype vulnerability in stem cell models of vanishing white matter

PS Leferink, S Dooves, AEJ Hillen… - Annals of …, 2019 - Wiley Online Library
Objective Astrocytes have gained attention as important players in neurological disease. In
line with their heterogeneous character, defects in specific astrocyte subtypes have been …

A ffected astrocytes in the spinal cord of the leukodystrophy vanishing white matter

PS Leferink, N Breeuwsma, M Bugiani… - Glia, 2018 - Wiley Online Library
Leukodystrophies are often devastating diseases, presented with progressive clinical signs
as spasticity, ataxia and cognitive decline, and lack proper treatment options. New therapy …

[HTML][HTML] Core protocol development for phase 2/3 clinical trials in the leukodystrophy vanishing white matter: a consensus statement by the VWM consortium and …

DH Schoenmakers, PS Leferink, A Vanderver… - BMC neurology, 2023 - Springer
Background The leukodystrophy “Vanishing White Matter” (VWM) is an orphan disease with
neurological decline and high mortality. Currently, VWM has no approved treatments, but …

[PDF][PDF] Cell replacement therapy improves pathological hallmarks in a mouse model of leukodystrophy vanishing white matter

S Dooves, PS Leferink, S Krabbenborg, N Breeuwsma… - Stem cell reports, 2019 - cell.com
Stem cell therapy has great prospects for brain white matter disorders, including the
genetically determined disorders called leukodystrophies. We focus on the devastating …

[HTML][HTML] The healthy and diseased microenvironments regulate oligodendrocyte properties: implications for regenerative medicine

PS Leferink, VM Heine - The American Journal of Pathology, 2018 - Elsevier
White matter disorders are characterized by deficient myelin or myelin loss, lead to a range
of neurologic dysfunctions, and can result in early death. Oligodendrocytes, which are …

Therapeutic potential of human stem cell transplantations for Vanishing White Matter: A quest for the Goldilocks graft

…, PS Leferink, NB Breeuwsma, S Dooves… - CNS Neuroscience …, 2022 - Wiley Online Library
Introduction Vanishing white matter (VWM) is a leukodystrophy that leads to neurological
dysfunction and early death. Astrocytes are indicated as therapeutic target, because of their …

Core protocol development for phase 2/3 clinical trials in the leukodystrophy Vanishing White Matter

DH Schoenmakers, PS Leferink, A Vanderver… - 2023 - researchsquare.com
Background: The leukodystrophy “Vanishing White Matter”(VWM) is an orphan disease with
neurological decline and high mortality. Currently, VWM has no approved treatments, but …