
OpenAlex is a bibliographic catalogue of scientific papers, authors and institutions accessible in open access mode, named after the Library of Alexandria. It's citation coverage is excellent and I hope you will find utility in this listing of citing articles!
If you click the article title, you'll navigate to the article, as listed in CrossRef. If you click the Open Access links, you'll navigate to the "best Open Access location". Clicking the citation count will open this listing for that article. Lastly at the bottom of the page, you'll find basic pagination options.
Requested Article:
Treatment of lysosomal storage disorders: successes and challenges
Carla E. M. Hollak, Frits A. Wijburg
Journal of Inherited Metabolic Disease (2014) Vol. 37, Iss. 4, pp. 587-598
Closed Access | Times Cited: 76
Carla E. M. Hollak, Frits A. Wijburg
Journal of Inherited Metabolic Disease (2014) Vol. 37, Iss. 4, pp. 587-598
Closed Access | Times Cited: 76
Showing 1-25 of 76 citing articles:
Pompe disease: from pathophysiology to therapy and back again
Jeong‐A Lim, Lishu Li, Nina Raben
Frontiers in Aging Neuroscience (2014) Vol. 6
Open Access | Times Cited: 177
Jeong‐A Lim, Lishu Li, Nina Raben
Frontiers in Aging Neuroscience (2014) Vol. 6
Open Access | Times Cited: 177
Lysosomal Re-acidification Prevents Lysosphingolipid-Induced Lysosomal Impairment and Cellular Toxicity
Christopher J. Folts, Nicole Scott‐Hewitt, Christoph Pröschel, et al.
PLoS Biology (2016) Vol. 14, Iss. 12, pp. e1002583-e1002583
Open Access | Times Cited: 68
Christopher J. Folts, Nicole Scott‐Hewitt, Christoph Pröschel, et al.
PLoS Biology (2016) Vol. 14, Iss. 12, pp. e1002583-e1002583
Open Access | Times Cited: 68
Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann–Pick disease type B (acid sphingomyelinase deficiency)
Margaret M. McGovern, Melissa P. Wasserstein, Brian Kirmse, et al.
Genetics in Medicine (2015) Vol. 18, Iss. 1, pp. 34-40
Open Access | Times Cited: 64
Margaret M. McGovern, Melissa P. Wasserstein, Brian Kirmse, et al.
Genetics in Medicine (2015) Vol. 18, Iss. 1, pp. 34-40
Open Access | Times Cited: 64
In vivo genome editing of mucopolysaccharidosis I mice using the CRISPR/Cas9 system
Roselena Silvestri Schuh, Édina Poletto, Gabriela Pasqualim, et al.
Journal of Controlled Release (2018) Vol. 288, pp. 23-33
Closed Access | Times Cited: 59
Roselena Silvestri Schuh, Édina Poletto, Gabriela Pasqualim, et al.
Journal of Controlled Release (2018) Vol. 288, pp. 23-33
Closed Access | Times Cited: 59
CNS-Targeting Therapies for Lysosomal Storage Diseases: Current Advances and Challenges
Mariola J. Edelmann, Gustavo Maegawa
Frontiers in Molecular Biosciences (2020) Vol. 7
Open Access | Times Cited: 55
Mariola J. Edelmann, Gustavo Maegawa
Frontiers in Molecular Biosciences (2020) Vol. 7
Open Access | Times Cited: 55
Exosome- and extracellular vesicle-based approaches for the treatment of lysosomal storage disorders
Biao Lü, Joy Ku, Renceh Flojo, et al.
Advanced Drug Delivery Reviews (2022) Vol. 188, pp. 114465-114465
Open Access | Times Cited: 30
Biao Lü, Joy Ku, Renceh Flojo, et al.
Advanced Drug Delivery Reviews (2022) Vol. 188, pp. 114465-114465
Open Access | Times Cited: 30
Failure to shorten the diagnostic delay in two ultra-orphan diseases (mucopolysaccharidosis types I and III): potential causes and implications
Gé‐Ann Kuiper, O. L. M. Meijer, Eveline J. Langereis, et al.
Orphanet Journal of Rare Diseases (2018) Vol. 13, Iss. 1
Open Access | Times Cited: 56
Gé‐Ann Kuiper, O. L. M. Meijer, Eveline J. Langereis, et al.
Orphanet Journal of Rare Diseases (2018) Vol. 13, Iss. 1
Open Access | Times Cited: 56
Multiplex Tandem Mass Spectrometry Enzymatic Activity Assay for Newborn Screening of the Mucopolysaccharidoses and Type 2 Neuronal Ceroid Lipofuscinosis
Liu Yang, Fan Yi, Arun Kumar, et al.
Clinical Chemistry (2017) Vol. 63, Iss. 6, pp. 1118-1126
Open Access | Times Cited: 50
Liu Yang, Fan Yi, Arun Kumar, et al.
Clinical Chemistry (2017) Vol. 63, Iss. 6, pp. 1118-1126
Open Access | Times Cited: 50
The Changing Landscape of Fabry Disease
Einar Svarstad, Hans‐Peter Marti
Clinical Journal of the American Society of Nephrology (2020) Vol. 15, Iss. 4, pp. 569-576
Open Access | Times Cited: 49
Einar Svarstad, Hans‐Peter Marti
Clinical Journal of the American Society of Nephrology (2020) Vol. 15, Iss. 4, pp. 569-576
Open Access | Times Cited: 49
Prevalence rates of mucopolysaccharidoses in Poland
Agnieszka Jurecka, Agnieszka Ługowska, Adam Gołda, et al.
Journal of Applied Genetics (2014) Vol. 56, Iss. 2, pp. 205-210
Open Access | Times Cited: 48
Agnieszka Jurecka, Agnieszka Ługowska, Adam Gołda, et al.
Journal of Applied Genetics (2014) Vol. 56, Iss. 2, pp. 205-210
Open Access | Times Cited: 48
Modulation of expression of genes involved in glycosaminoglycan metabolism and lysosome biogenesis by flavonoids
Marta Moskot, Joanna Jakóbkiewicz‐Banecka, Anna Kłoska, et al.
Scientific Reports (2015) Vol. 5, Iss. 1
Open Access | Times Cited: 47
Marta Moskot, Joanna Jakóbkiewicz‐Banecka, Anna Kłoska, et al.
Scientific Reports (2015) Vol. 5, Iss. 1
Open Access | Times Cited: 47
The treatment of juvenile/adult GM1-gangliosidosis with Miglustat may reverse disease progression
Federica Deodato, Elena Procopio, Angelica Rampazzo, et al.
Metabolic Brain Disease (2017) Vol. 32, Iss. 5, pp. 1529-1536
Closed Access | Times Cited: 44
Federica Deodato, Elena Procopio, Angelica Rampazzo, et al.
Metabolic Brain Disease (2017) Vol. 32, Iss. 5, pp. 1529-1536
Closed Access | Times Cited: 44
Acid ceramidase and the treatment of ceramide diseases: The expanding role of enzyme replacement therapy
Edward H. Schuchman
Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease (2016) Vol. 1862, Iss. 9, pp. 1459-1471
Closed Access | Times Cited: 41
Edward H. Schuchman
Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease (2016) Vol. 1862, Iss. 9, pp. 1459-1471
Closed Access | Times Cited: 41
Enzyme replacement therapy for Farber disease: Proof-of-concept studies in cells and mice
Xingxuan He, Shaalee Dworski, Changzhi Zhu, et al.
BBA Clinical (2017) Vol. 7, pp. 85-96
Open Access | Times Cited: 41
Xingxuan He, Shaalee Dworski, Changzhi Zhu, et al.
BBA Clinical (2017) Vol. 7, pp. 85-96
Open Access | Times Cited: 41
Disease models for the development of therapies for lysosomal storage diseases
Miao Xu, Omid Motabar, Marc Ferrer, et al.
Annals of the New York Academy of Sciences (2016) Vol. 1371, Iss. 1, pp. 15-29
Open Access | Times Cited: 40
Miao Xu, Omid Motabar, Marc Ferrer, et al.
Annals of the New York Academy of Sciences (2016) Vol. 1371, Iss. 1, pp. 15-29
Open Access | Times Cited: 40
‘Fighting every day’: exploring caregiver quality of life and perspectives on healthcare services for children with dementia – a cross-sectional, mixed-methods study
Jason V. Djafar, Suzanne M. Nevin, Nicholas Smith, et al.
Archives of Disease in Childhood (2025), pp. archdischild-328011
Closed Access
Jason V. Djafar, Suzanne M. Nevin, Nicholas Smith, et al.
Archives of Disease in Childhood (2025), pp. archdischild-328011
Closed Access
Gene editing of MPS I human fibroblasts by co-delivery of a CRISPR/Cas9 plasmid and a donor oligonucleotide using nanoemulsions as nonviral carriers
Roselena Silvestri Schuh, Talita Giacomet de Carvalho, Roberto Giugliani, et al.
European Journal of Pharmaceutics and Biopharmaceutics (2017) Vol. 122, pp. 158-166
Closed Access | Times Cited: 39
Roselena Silvestri Schuh, Talita Giacomet de Carvalho, Roberto Giugliani, et al.
European Journal of Pharmaceutics and Biopharmaceutics (2017) Vol. 122, pp. 158-166
Closed Access | Times Cited: 39
Extraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression
Martin L. Katz, G. C. Johnson, Stacey B. Leach, et al.
Gene Therapy (2017) Vol. 24, Iss. 4, pp. 215-223
Open Access | Times Cited: 35
Martin L. Katz, G. C. Johnson, Stacey B. Leach, et al.
Gene Therapy (2017) Vol. 24, Iss. 4, pp. 215-223
Open Access | Times Cited: 35
Lysosomal Storage Disorders in Egyptian Children
Mohamed A. Elmonem, Iman G. Mahmoud, Dina Mehaney, et al.
The Indian Journal of Pediatrics (2016) Vol. 83, Iss. 8, pp. 805-813
Closed Access | Times Cited: 31
Mohamed A. Elmonem, Iman G. Mahmoud, Dina Mehaney, et al.
The Indian Journal of Pediatrics (2016) Vol. 83, Iss. 8, pp. 805-813
Closed Access | Times Cited: 31
Emerging therapies for neuropathic lysosomal storage disorders
Jessica M. Kelly, Allison M. Bradbury, Douglas R. Martin, et al.
Progress in Neurobiology (2016) Vol. 152, pp. 166-180
Closed Access | Times Cited: 31
Jessica M. Kelly, Allison M. Bradbury, Douglas R. Martin, et al.
Progress in Neurobiology (2016) Vol. 152, pp. 166-180
Closed Access | Times Cited: 31
Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Mucopolysaccharidoses
Valeria De Pasquale, Patrizia Sarogni, Valéria Pistorio, et al.
Molecular Therapy — Methods & Clinical Development (2018) Vol. 10, pp. 8-16
Open Access | Times Cited: 31
Valeria De Pasquale, Patrizia Sarogni, Valéria Pistorio, et al.
Molecular Therapy — Methods & Clinical Development (2018) Vol. 10, pp. 8-16
Open Access | Times Cited: 31
Newborn Screening for Lysosomal Storage Disorders
Roy W A Peake, Olaf A. Bodamer
Journal of Pediatric Genetics (2016) Vol. 06, Iss. 01, pp. 051-060
Open Access | Times Cited: 30
Roy W A Peake, Olaf A. Bodamer
Journal of Pediatric Genetics (2016) Vol. 06, Iss. 01, pp. 051-060
Open Access | Times Cited: 30
Safety Evaluation of CNS Administered Biologics—Study Design, Data Interpretation, and Translation to the Clinic
Brian R. Vuillemenot, Sven Korte, Teresa L. Wright, et al.
Toxicological Sciences (2016) Vol. 152, Iss. 1, pp. 3-9
Open Access | Times Cited: 29
Brian R. Vuillemenot, Sven Korte, Teresa L. Wright, et al.
Toxicological Sciences (2016) Vol. 152, Iss. 1, pp. 3-9
Open Access | Times Cited: 29
Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice
Roselena Silvestri Schuh, Esteban Alberto Gonzalez, Ângela Maria Vicente Tavares, et al.
Gene Therapy (2019) Vol. 27, Iss. 1-2, pp. 74-84
Open Access | Times Cited: 28
Roselena Silvestri Schuh, Esteban Alberto Gonzalez, Ângela Maria Vicente Tavares, et al.
Gene Therapy (2019) Vol. 27, Iss. 1-2, pp. 74-84
Open Access | Times Cited: 28
Nanotechnology applied to treatment of mucopolysaccharidoses
Roselena Silvestri Schuh, Guilherme Baldo, Hélder Ferreira Teixeira
Expert Opinion on Drug Delivery (2016) Vol. 13, Iss. 12, pp. 1709-1718
Closed Access | Times Cited: 26
Roselena Silvestri Schuh, Guilherme Baldo, Hélder Ferreira Teixeira
Expert Opinion on Drug Delivery (2016) Vol. 13, Iss. 12, pp. 1709-1718
Closed Access | Times Cited: 26