Results 1 to 10 of about 4,879,561 (170)

Nociceptive pain in late-onset Pompe disease: prevalence, distribution, and clinical correlates. [PDF]

J Neurol
Wenninger S, Arndt M, Mendelsohn DH, Wirner-Piotrowski C, Mijic M, Garcia-Angarita N, Gutschmidt K, Schmidt C, Winkler L, Gläser D, Knop KC, Spranger M, Klawonn F, Brauner LE, Schoser B.   +14 more
europepmc   +1 more source

Defining the therapeutic corridor of stability in enzyme replacement therapy for Pompe disease: a position statement. [PDF]

Orphanet J Rare Dis
Schoser B.
europepmc   +1 more source

A Comprehensive Update on Pompe Disease: From Existing Therapies to Emerging Curative Strategies. [PDF]

Int J Mol Sci
Estevez Barcia R, Colón C, Hermida-Ameijeiras Á, López-Valverde L, Rodrigues D, Domínguez-González C, Díaz-Manera J, Couce ML, Alvarez JV.   +8 more
europepmc   +1 more source

Identification of alterations of immunometabolism associated with Pompe disease. [PDF]

Cell Mol Life Sci
Costa-Verdera H, Gargaro M, Cagin U, Manni G, Scalisi G, Veron P, Silva Barcelos EC, Pieroni B, Mencarelli G, Ricciuti D, Nemazanyy I, Sanatine P, van Wittenberghe L, Laforêt P, Gross DA, Ronzitti G, Fallarino F, Mingozzi F.   +17 more
europepmc   +1 more source

Cardiac magnetic resonance in Pompe disease: a systematic literature review. [PDF]

Radiol Med
Lupi A, Schiavone E, Gragnaniello V, Cervaro S, Crimì F, Quaia E, Burlina A, Pepe A.   +7 more
europepmc   +1 more source

Central nervous system histopathological findings in classic infantile Pompe disease: a systematic review with clinical relevance. [PDF]

J Neurol
van den Dorpel JJA, Faraguna MC, Verdijk RM, van der Beek NAME, Pijnappel WWMP, van der Ploeg AT, van den Hout JMP.   +6 more
europepmc   +1 more source

Managing Pompe Disease and Enzyme Replacement Therapy During Pregnancy: Challenges and Considerations. [PDF]

Eur J Neurol
Theunissen MTM, Hayat Z, Bouhour F, van den Hout JMP, Noury JB, Lagrange E, Jouy A, Taouagh N, van Doorn PA, van der Ploeg AT, Laforêt P, van der Beek NAME.   +11 more
europepmc   +1 more source

Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease. [PDF]

Mol Ther
Fuller DD, Rana S, Thakre PP, Benevides ES, Pope MK, Todd AG, Jensen VN, Vaught L, Cloutier DA, Ribas RA, Larson RC, Gentry MS, Sun RC, Chandran V, Corti M, Falk DJ, Byrne BJ.   +16 more
europepmc   +1 more source

A roadmap for a patient-centred approach to Pompe disease management. [PDF]

J Neurol
Schoser B, Domínguez-González C, Laforet P, Hahn A, Gissen P, Kostera-Pruszczyk A, Thalmeier A, Vissing J.   +7 more
europepmc   +1 more source

Cost-Effectiveness of Newborn Screening for Infantile-Onset Pompe Disease in Japan. [PDF]

Int J Neonatal Screen
Konomura K, Tanaka M, Tajima G, Hoshino E.   +3 more
europepmc   +1 more source