Results 91 to 100 of about 275,755 (180)

Gastrointestinal manifestations and enzyme replacement therapy in late-onset Pompe disease: insights from a cross-sectional analysis. [PDF]

Orphanet J Rare Dis
Liu X, Li X, Yao X, Zhu G, Guo X, He W, Hu L, Yang G.   +7 more
europepmc   +1 more source

Early Initiation of Enzyme Replacement Therapy in Infantile Onset Pompe Disease Improves Cardiac Outcomes: A Longitudinal Analysis. [PDF]

JIMD Rep
Cohen JL, Beaman MM, Rodriguez-Rassi E, Stafford VG, Smith PB, Landstrom AP, Kishnani PS.   +6 more
europepmc   +1 more source

Integration of ERT and Geotechnical Investigation for River Restoration: A Case Study of Dam Removal Site Characterization. [PDF]

Environ Manage
Rahimi M, Wood CM, Befus KM, Holt JJ, Thompson G, Fathizadeh M.   +5 more
europepmc   +1 more source

Efficacy and Safety of Epidermal Radionuclide Therapy with ¹⁸⁸Re Resin in Patients with Nonmelanoma Skin Cancers: Long-Term Data from a Single Center. [PDF]

J Nucl Med
Castellucci P, Baraldi C, Vetrone L, Zagni F, Malizia C, Morganti AG, Strigari L, Dika E, Fanti S.   +8 more
europepmc   +1 more source

Insights into immunogenicity and therapeutic strategies to mitigate the immune response in infantile-onset Pompe disease: a comprehensive systematic literature review. [PDF]

Front Immunol
Kishnani PS, Van Den Hout JMP, Hahn A, Kronn D, Chien YH, Han M, Heuterman J, Sparks S, Glen C, Daba N.   +9 more
europepmc   +1 more source

Comprehensive review of recent advances in Pompe disease: pathogenesis, management, and future directions. [PDF]

Front Neurol
Li G.
europepmc   +1 more source

Assessment of liver and spleen stiffness and hepatic steatosis by transient elastography (Fibroscan®) in type 1 Gaucher disease: a single center case-control cohort study. [PDF]

Orphanet J Rare Dis
Mutlu U, Cavus B, Hacisahinogullari H, Yenidunya Yalin G, Soyluk Selcukbiricik O, Gul N, Kubat Uzum A, Demir K, Tanakol R.   +8 more
europepmc   +1 more source

ERT

Indústrias Culturais, 2013
openaire   +1 more source

Enzyme replacement therapy during pregnancy and breastfeeding in late-onset Pompe disease. [PDF]

Int Breastfeed J
Bachmann M, Kasper D, Scholl-Bürgi S, Margreitter J, Höller A, Löscher W, Karall D, Zöggeler T.   +7 more
europepmc   +1 more source

Exemplifying a measurement validation strategy for rare- and ultra-rare diseases: measuring what matters in alpha-mannosidosis. [PDF]

Orphanet J Rare Dis
Schwartz CE, Borowiec K, Koulinska I, Morgan H, Rapkin BD.   +4 more
europepmc   +1 more source