Long-term prognosis of patients with infantile-onset Pompe disease diagnosed by newborn screening and treated since birth.

Long-term prognosis of patients with infantile-onset Pompe disease diagnosed by newborn screening and treated since birth.

Item
http://www.wikidata.org/entity/Q50608296
Albuterol as an adjunctive treatment to enzyme replacement therapy in infantile-onset Pompe disease.Liquid Chromatography-Tandem Mass Spectrometry Assay of Leukocyte Acid α-Glucosidase for Post-Newborn Screening Evaluation of Pompe Disease.Slow, progressive myopathy in neonatally treated patients with infantile-onset Pompe disease: a muscle magnetic resonance imaging study.Divergent clinical outcomes of alpha-glucosidase enzyme replacement therapy in two siblings with infantile-onset Pompe disease treated in the symptomatic or pre-symptomatic state.Preventing pediatric cardiomyopathy: a 2015 outlook.Erythrocyte-mediated delivery of recombinant enzymes.A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice.The investigation and management of metabolic myopathies.Survey of Italian pediatricians' perspectives and knowledge about neonatal screening.Neuroimaging findings in infantile Pompe patients treated with enzyme replacement therapy.Lysosomal Storage Disorders: Present and Future.Infantile-onset Pompe disease with neonatal debut: A case report and literature review.Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy.Cognitive and academic outcomes in long-term survivors of infantile-onset Pompe disease: A longitudinal follow-up.
P2860

Long-term prognosis of patients with infantile-onset Pompe disease diagnosed by newborn screening and treated since birth.

Item
http://www.wikidata.org/entity/Q50608296
description
2014 nî lūn-bûn
@nan
2014年の論文
@ja
2014年学术文章
@wuu
2014年学术文章
@zh
2014年学术文章
@zh-cn
2014年学术文章
@zh-hans
2014年学术文章
@zh-my
2014年学术文章
@zh-sg
2014年學術文章
@yue
2014年學術文章
@zh-hant
name
Long-term prognosis of patient ...... ening and treated since birth.
@en
Long-term prognosis of patient ...... ening and treated since birth.
@nl
type
label
Long-term prognosis of patient ...... ening and treated since birth.
@en
Long-term prognosis of patient ...... ening and treated since birth.
@nl
prefLabel
Long-term prognosis of patient ...... ening and treated since birth.
@en
Long-term prognosis of patient ...... ening and treated since birth.
@nl
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Long-term prognosis of patient ...... eening and treated since birth
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P2093
P304
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P356
10.1016/J.JPEDS.2014.10.068
P407
P433
P478
P50
P577
P698