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Journal of Human Genetics|April 25, 2019
SOPH syndrome in three affected individuals showing similarities with progeroid cutis laxa conditions in early infancyBjörn Fischer-Zirnsak, Rainer Koenig, Franz Alisch, et al.
Journal of Hepatology|May 7, 2016
Severe respiratory complex III defect prevents liver adaptation to prolonged fastingLaura S Kremer, Caroline L'hermitte-Stead, Pierre Lesimple, et al.
Annals of Clinical and Translational Neurology|October 1, 2025
The Diverse Neuromuscular Spectrum of VPS13A DiseaseAnne Buchberger, Evamaria Riedel, Marie Hackenberg, et al.
Neurological Sciences : Official Journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology|August 22, 2018
Clinical presentation and natural history of infantile-onset ascending spastic paralysis from three families with an ALS2 founder variantMayada Helal, Neda Mazaheri, Bita Shalbafan, et al.
Clinical Genetics|June 24, 2021
Expanding the phenotypic spectrum of FINCA (fibrosis, neurodegeneration, and cerebral angiomatosis) syndrome beyond infancyChristina K Rapp, Ine Van Dijck, Lucia Laugwitz, et al.
JIMD Reports|April 18, 2014
Common and Novel TMEM70 Mutations in a Cohort of Italian Patients with Mitochondrial EncephalocardiomyopathyDaria Diodato, Federica Invernizzi, Eleonora Lamantea, et al.
Journal of Neurology|July 20, 2024
Novel variants in CSF1R associated with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)Anne S Schmitz, Janani Raju, Wolfgang Köhler, et al.
Frontiers in Cell and Developmental Biology|February 2, 2023
PHIP-associated Chung-Jansen syndrome: Report of 23 new individualsAntje Kampmeier, Elsa Leitão, Ilaria Parenti, et al.
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