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Stem Cell Reports|September 29, 2023
Highly contractile 3D tissue engineered skeletal muscles from human iPSCs reveal similarities with primary myoblast-derived tissuesErik van der Wal, Alessandro Iuliano, Stijn L M In 't Groen, et al.
Molecular Therapy. Methods & Clinical Development|June 6, 2022
Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe diseaseQiushi Liang, Eva C Vlaar, Fabio Catalano, et al.
Human Mutation|August 18, 2021
Broad variation in phenotypes for common GAA genotypes in Pompe diseaseMonica Y Niño, Stijn L M In't Groen, Douglas O S de Faria, et al.
Molecular Therapy. Nucleic Acids|June 19, 2017
GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle CellsErik van der Wal, Atze J Bergsma, Tom J M van Gestel, et al.
Journal of Tissue Engineering|May 4, 2026
Fibro-adipogenic progenitors enhance functional and structural properties of human 3D tissue engineered skeletal musclesRoy Augustinus, Lotte A de Ridder, Dongxu Zheng, et al.
Archives of Physical Medicine and Rehabilitation|December 16, 2014
Exercise training in adults with Pompe disease: the effects on pain, fatigue, and functioningMarein M Favejee, Linda E M van den Berg, Michelle E Kruijshaar, et al.
Journal of Inherited Metabolic Disease|June 8, 2020
Effects of higher and more frequent dosing of alglucosidase alfa and immunomodulation on long-term clinical outcome of classic infantile Pompe patientsEsther Poelman, Jan J A van den Dorpel, Marianne Hoogeveen-Westerveld, et al.
Orphanet Journal of Rare Diseases|February 3, 2022
Antibodies against recombinant human alpha-glucosidase do not seem to affect clinical outcome in childhood onset Pompe diseaseHarmke A van Kooten, Imke A M Ditters, Marianne Hoogeveen-Westerveld, et al.
Stem Cell Reports|May 8, 2018
Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic StrategiesErik van der Wal, Pablo Herrero-Hernandez, Raymond Wan, et al.
Biodrugs : Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy|November 24, 2025
Can Alpha-Glucosidase Activity in Plasma or Leukocytes Serve as a Biomarker for Future Gene Therapy in Classic Infantile Pompe Disease?Martha C Faraguna, Daniël A M Lambregts, Ina Barzel, et al.
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