Latest Research in Lysosomal Storage Disorders Research

34 research papers · 2026 median publication year

Top Research Topics in Lysosomal Storage Disorders Research

Highest-Cited Papers

  1. Tangier Disease
  2. Cramps as the initial presentation of CANVAS/RFC1-related disorder
  3. Late onset biotinidase deficiency presenting with optic neuropathy and multifocal motor neuropathy phenotype: a case report and literature review
  4. Involvement of Reduced ENPP1 Function in the Pathogenesis of Ossification of the Posterior Longitudinal Ligament
  5. Plasma autoantibodies as novel risk markers for stroke: A nested case-control study of the JPHC study
  6. Progressive dilated cardiomyopathy in a patient with previously unrecognized Klinefelter syndrome: a case report
  7. Genetic Spectrum of Cholestasis in Tunisia and Diagnostic Yield of Next‐Generation Sequencing: Case Series of 70 Patients
  8. Beyond Sequencing: Integrating MLPA Reveals Hidden Structural PKD2 Variants and Enhances Mutation Detection in a Highly Selected ADPKD Greek Cohort
  9. Tissue-nonspecific alkaline phosphatase deficiency is associated with altered immune cell profiles in hypophosphatasia
  10. Timely diagnosis of early-onset forms of metachromatic leukodystrophy: an Italian consensus study
  11. Genetic Testing Unveils a Novel Thrombospondin‐1 Domain Containing Protein 1 Gene Variant as the Cause of Chronic Edema in a 79‐Year‐Old Woman
  12. Glycoproteomics and Functional Characterization of Novel Variants in Siblings with ALG1-CDG
  13. A Combination of Alleles in LMOD2 and a lncRNA is Strongly Associated With Myxomatous Mitral Valve Disease in Cavalier King Charles Spaniels
  14. Comment on the Cerebral Astrocytic Inclusion Pathology in Multiple System Atrophy With a Long Clinical Course
  15. Clinical Variability of Classical Ehlers–Danlos Syndrome: A Family with Rare COL5A1 Variant and Case-Based Review
  16. Urine-Derived Cells in Kidney Transplantation: Linking Cellular Phenotypes, Secretome Signatures and Multi-Omic Technologies
  17. Autoimmune pernicious anemia in clinically diagnosed Bardet–Biedl syndrome: a case report and brief literature review
  18. Treatment updates in myotonic disorders
  19. Fabry Disease: Integrating Molecular Pathophysiology, Precision Diagnosis, and Artificial Intelligence Toward Precision Medicine
  20. Role of Local Growth Plate Mechanisms and Systemic Endocrine Signals in X-Linked Hypophosphatemia (XLH) Impaired Linear Growth
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L3 Region - - 2026 Sep Q3

Lysosomal Storage Disorders Research

34 papers

Top Topics (10)

Lysosomal Storage Disorders Research3
Genetic Neurodegenerative Diseases2
Genetic and Kidney Cyst Diseases2
Alkaline Phosphatase Research Studies2
Connective tissue disorders research2
Myasthenia Gravis and Thymoma2
Cholesterol and Lipid Metabolism1
Biotin and Related Studies1
Cervical and Thoracic Myelopathy1
Atherosclerosis and Cardiovascular Diseases1

Top Publications (20)

1.Tangier Disease2.Cramps as the initial presentation of CANVAS/RFC1-related disorder3.Late onset biotinidase deficiency presenting with optic neuropathy and multifocal motor neuropathy phenotype: a case report and literature review4.Involvement of Reduced ENPP1 Function in the Pathogenesis of Ossification of the Posterior Longitudinal Ligament5.Plasma autoantibodies as novel risk markers for stroke: A nested case-control study of the JPHC study6.Progressive dilated cardiomyopathy in a patient with previously unrecognized Klinefelter syndrome: a case report7.Genetic Spectrum of Cholestasis in Tunisia and Diagnostic Yield of Next‐Generation Sequencing: Case Series of 70 Patients8.Beyond Sequencing: Integrating MLPA Reveals Hidden Structural PKD2 Variants and Enhances Mutation Detection in a Highly Selected ADPKD Greek Cohort9.Tissue-nonspecific alkaline phosphatase deficiency is associated with altered immune cell profiles in hypophosphatasia10.Timely diagnosis of early-onset forms of metachromatic leukodystrophy: an Italian consensus study11.Genetic Testing Unveils a Novel Thrombospondin‐1 Domain Containing Protein 1 Gene Variant as the Cause of Chronic Edema in a 79‐Year‐Old Woman12.Glycoproteomics and Functional Characterization of Novel Variants in Siblings with ALG1-CDG13.A Combination of Alleles in LMOD2 and a lncRNA is Strongly Associated With Myxomatous Mitral Valve Disease in Cavalier King Charles Spaniels14.Comment on the Cerebral Astrocytic Inclusion Pathology in Multiple System Atrophy With a Long Clinical Course15.Clinical Variability of Classical Ehlers–Danlos Syndrome: A Family with Rare COL5A1 Variant and Case-Based Review16.Urine-Derived Cells in Kidney Transplantation: Linking Cellular Phenotypes, Secretome Signatures and Multi-Omic Technologies17.Autoimmune pernicious anemia in clinically diagnosed Bardet–Biedl syndrome: a case report and brief literature review18.Treatment updates in myotonic disorders19.Fabry Disease: Integrating Molecular Pathophysiology, Precision Diagnosis, and Artificial Intelligence Toward Precision Medicine20.Role of Local Growth Plate Mechanisms and Systemic Endocrine Signals in X-Linked Hypophosphatemia (XLH) Impaired Linear Growth
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