Results 161 to 170 of about 22,388 (216)

Elevated Transaminases: Does It Always Warrant a Liver Biopsy? Lessons Learned From Pompe Disease. [PDF]

JIMD Rep
Khazzeka A, Koch RL, Lim JA, Ward JD, Doxey J, Jeck WR, Kishnani PS.   +6 more
europepmc   +1 more source

Correction: Can Alpha-Glucosidase Activity in Plasma or Leukocytes Serve as a Biomarker for Future Gene Therapy in Classic Infantile Pompe Disease? [PDF]

BioDrugs
Faraguna MC, Lambregts DAM, Barzel I, Jacobs EH, Hoogeveen-Westerveld M, van der Beek NAME, Pijnappel WWMP, Gasperini S, Preijers T, van den Hout JMP, van der Ploeg AT.   +10 more
europepmc   +1 more source

Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis.

Health Technol Assess
Corbett M, Umemneku-Chikere C, Nevitt S, Deng NJ, Walton M, Fulbright H, Tan CY, Lachmann R, Churchill R, Hodgson R.   +9 more
europepmc   +1 more source

Anaesthetic Management of Advanced Late-Onset Pompe Disease: Challenges in a Major Abdominal Surgery. [PDF]

Cureus
Silva M, Santos MF, Cardoso G.
europepmc   +1 more source

Bone disease and osteoporosis associated with Pompe disease. [PDF]

JBMR Plus
Maxey L, Freibert H, Quinonez A, Malluche H, Rao M.   +4 more
europepmc   +1 more source

Atrioventricular Block in a Pompe Disease Patient Receiving Enzyme Replacement Therapy. [PDF]

JACC Case Rep
Akagi K, Baba S, Matsuda K, Yoshida T, Hirata T, Yokoyama A, Yoneda F, Fukumura F, Kume E, Takita J.   +9 more
europepmc   +1 more source

Plasma glial fibrillary acidic protein (GFAP) is a biomarker for central nervous system involvement in infantile-onset Pompe disease. [PDF]

EBioMedicine
Regmi N, Abar B, Lim JA, Kenney-Jung D, Malinzak M, Hagarty-Waite KA, Jones KA, Jung SH, Stiles AR, Koch RL, Kishnani PS.   +10 more
europepmc   +1 more source

Causes of Death and Comorbidities in Adult Patients With Late-Onset Pompe Disease: A French Pompe Registry Retrospective Study. [PDF]

Eur J Neurol
Chitimus DM, Tard C, Fournier M, Bouhour F, Béhin A, Salort-Campana E, Lagrange E, Kaminsky AL, Magot A, Beltran S, Noury JB, Magy L, Solé G, Renard D, Spinazzi M, Demurger F, Cintas P, Nadaj-Pakleza A, Deibener-Kaminsky J, Bassez G, Taouagh N, Arrassi A, Lefeuvre C, Attarian S, Hamroun D, Laforêt P, for French Pompe study group.   +26 more
europepmc   +1 more source

Latest Advancements in Treatment Options for Infantile-Onset Pompe Disease: A Comprehensive Narrative Review. [PDF]

Clin Med Insights Pediatr
Khan M, Ibrahim M, Riaz F, Awan Z, Zulfiqar E, Kumar A, Al Sakini AS, Khan TM, Al-Shammari AS.   +8 more
europepmc   +1 more source