Safety and efficacy of exercise training in adults with Pompe disease: evalution of endurance, muscle strength and core stability before and after a 12 week training program [PDF]
, 2015 Linda E.M. van den Berg +8 more
openalex +1 more source
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations [PDF]
John K. Yoon +27 more
openalex +1 more source
Identification of miRNAs Associated with Infantile-Onset Pompe Disease. [PDF]
Mol SyndromolBayrak H, Tosun F.
europepmc +1 more source
Development of Newborn Screening for Pompe Disease [PDF]
, 2020 Wuh‐Liang Hwu, Yin‐Hsiu Chien
openalex +1 more source
P029: Early treatment with alglucosidase alfa is associated with improved survival in patients with infantile-onset Pompe disease: Data from Pompe Registry [PDF]
Priya S. Kishnani +10 more
openalex +1 more source
In Thickness and in Health: Delayed-Onset Pompe Disease Resembling Hypertrophic Cardiomyopathy. [PDF]
CureusLasam G, Lasam MKC.
europepmc +1 more source
Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease [PDF]
, 2014 Carin M. Van Gelder +5 more
openalex +1 more source
Molecular characterization of a novel synonymous variant in a Mexican patient with Pompe disease. [PDF]
Mol Genet Metab RepAlaez-Verson C +9 more
europepmc +1 more source
Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data [PDF]
Priya S. Kishnani +9 more
openalex +1 more source