Chen H, Hsu R, Fang C, Desai A, Lee N, Hwu W
    
    
    Front Immunol. 2024; 15:1336599.
  
  
    PMID: 38715621
    
          PMC: 11074348.
    
          DOI: 10.3389/fimmu.2024.1336599.
      
 
                                  
  
    Inci A, Ezgu F, Tumer L
    
    
    Paediatr Drugs. 2024; 26(3):287-308.
  
  
    PMID: 38664313
    
          PMC: 11074017.
    
          DOI: 10.1007/s40272-024-00627-9.
      
 
                                  
  
    Pfrimmer C, Smitka M, Muschol N, Husain R, Huemer M, Hennermann J
    
    
    J Neuromuscul Dis. 2023; 11(1):167-177.
  
  
    PMID: 38043017
    
          PMC: 10789365.
    
          DOI: 10.3233/JND-230164.
      
 
                                  
  
    Kistner A, Chichester J, Wang L, Calcedo R, Greig J, Cardwell L
    
    
    Gene Ther. 2023; 31(3-4):128-143.
  
  
    PMID: 37833563
    
          PMC: 10940161.
    
          DOI: 10.1038/s41434-023-00423-z.
      
 
                                  
  
    Ditters I, van Kooten H, van der Beek N, van der Ploeg A, Huidekoper H, van den Hout J
    
    
    Biomolecules. 2023; 13(9).
  
  
    PMID: 37759814
    
          PMC: 10526476.
    
          DOI: 10.3390/biom13091414.
      
 
                              
              
                              
                                      
  Monitoring and Management of Respiratory Function in Pompe Disease: Current Perspectives.
  
    El Haddad L, Khan M, Soufny R, Mummy D, Driehuys B, Mansour W
    
    
    Ther Clin Risk Manag. 2023; 19:713-729.
  
  
    PMID: 37680303
    
          PMC: 10480292.
    
          DOI: 10.2147/TCRM.S362871.
      
 
                                          
                                                          
  Long-Term Experience with Anaphylaxis and Desensitization to Alglucosidase Alfa in Pompe Disease.
  
    Ertoy Karagol H, Inci A, Polat Terece S, Kilic A, Demir F, Yapar D
    
    
    Int Arch Allergy Immunol. 2023; 184(4):370-375.
  
  
    PMID: 36623499
    
          PMC: 10137310.
    
          DOI: 10.1159/000528343.
      
 
                                          
                                                          
  Oral tolerance to prevent anti-drug antibody formation in protein replacement therapies.
  
    Rana J, Munoz M, Biswas M
    
    
    Cell Immunol. 2022; 382:104641.
  
  
    PMID: 36402002
    
          PMC: 9730862.
    
          DOI: 10.1016/j.cellimm.2022.104641.
      
 
                                          
                                                          
  The Clinical Management of Pompe Disease: A Pediatric Perspective.
  
    Marques J
    
    
    Children (Basel). 2022; 9(9).
  
  
    PMID: 36138713
    
          PMC: 9497581.
    
          DOI: 10.3390/children9091404.
      
 
                                          
                                                          
  A Multi-Centre Prospective Study of the Efficacy and Safety of Alglucosidase Alfa in Chinese Patients With Infantile-Onset Pompe Disease.
  
    Zhu D, Zhu J, Qiu W, Wang B, Liu L, Yu X
    
    
    Front Pharmacol. 2022; 13:903488.
  
  
    PMID: 35833019
    
          PMC: 9271607.
    
          DOI: 10.3389/fphar.2022.903488.
      
 
                                          
                                                          
  Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease.
  
    Liang Q, Vlaar E, Catalano F, Pijnenburg J, Stok M, van Helsdingen Y
    
    
    Mol Ther Methods Clin Dev. 2022; 25:520-532.
  
  
    PMID: 35662813
    
          PMC: 9127119.
    
          DOI: 10.1016/j.omtm.2022.04.016.
      
 
                                          
                                                          
  What's new and what's next for gene therapy in Pompe disease?.
  
    Roger A, Sethi R, Huston M, Scarrow E, Bao-Dai J, Lai E
    
    
    Expert Opin Biol Ther. 2022; 22(9):1117-1135.
  
  
    PMID: 35428407
    
          PMC: 10084869.
    
          DOI: 10.1080/14712598.2022.2067476.
      
 
                                          
                                                          
  To detect potential pathways and target genes in infantile Pompe patients using computational analysis.
  
    Karadag Gurel A, Gurel S
    
    
    Bioimpacts. 2022; 12(2):89-105.
  
  
    PMID: 35411297
    
          PMC: 8905584.
    
          DOI: 10.34172/bi.2022.23467.
      
 
                                          
                                                          
  Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel.
  
    Gragnaniello V, Deodato F, Gasperini S, Donati M, Canessa C, Fecarotta S
    
    
    Ital J Pediatr. 2022; 48(1):41.
  
  
    PMID: 35248118
    
          PMC: 8898438.
    
          DOI: 10.1186/s13052-022-01219-4.
      
 
                                          
                                                          
  Mechanisms of Neutralizing Anti-drug Antibody Formation and Clinical Relevance on Therapeutic Efficacy of Enzyme Replacement Therapies in Fabry Disease.
  
    Lenders M, Brand E
    
    
    Drugs. 2021; 81(17):1969-1981.
  
  
    PMID: 34748189
    
          PMC: 8602155.
    
          DOI: 10.1007/s40265-021-01621-y.
      
 
                                          
                                                          
  Pain Phenotypes in Rare Musculoskeletal and Neuromuscular Diseases.
  
    Tucker-Bartley A, Lemme J, Gomez-Morad A, Shah N, Veliu M, Birklein F
    
    
    Neurosci Biobehav Rev. 2021; 124:267-290.
  
  
    PMID: 33581222
    
          PMC: 9521731.
    
          DOI: 10.1016/j.neubiorev.2021.02.009.
      
 
                                          
                                                          
  Immunogenicity Challenges Associated with Subcutaneous Delivery of Therapeutic Proteins.
  
    Jarvi N, Balu-Iyer S
    
    
    BioDrugs. 2021; 35(2):125-146.
  
  
    PMID: 33523413
    
          PMC: 7848667.
    
          DOI: 10.1007/s40259-020-00465-4.
      
 
                                          
                                                          
  Transforming the clinical outcome in CRIM-negative infantile Pompe disease identified via newborn screening: the benefits of early treatment with enzyme replacement therapy and immune tolerance induction.
  
    Li C, Desai A, Gupta P, Dempsey K, Bhambhani V, Hopkin R
    
    
    Genet Med. 2021; 23(5):845-855.
  
  
    PMID: 33495531
    
          PMC: 8107133.
    
          DOI: 10.1038/s41436-020-01080-y.
      
 
                                          
                                                          
  Expansion of immature, nucleated red blood cells by transient low-dose methotrexate immune tolerance induction in mice.
  
    Tran J, Grover D, Zhang M, Stapels M, Brennan R, Bangari D
    
    
    Clin Exp Immunol. 2020; 203(3):409-423.
  
  
    PMID: 33205401
    
          PMC: 7874831.
    
          DOI: 10.1111/cei.13552.
      
 
                                          
                                                          
  Newborn Screening for Pompe Disease: Pennsylvania Experience.
  
    Ficicioglu C, Ahrens-Nicklas R, Barch J, Cuddapah S, DiBoscio B, DiPerna J
    
    
    Int J Neonatal Screen. 2020; 6(4).
  
  
    PMID: 33202836
    
          PMC: 7712483.
    
          DOI: 10.3390/ijns6040089.