Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years.
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The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?Quantification of Diaphragm Mechanics in Pompe Disease Using Dynamic 3D MRIRespiratory function in late-onset Pompe disease patients receiving long-term enzyme replacement therapy for more than 48 months.Muscle imaging data in late-onset Pompe disease reveal a correlation between the pre-existing degree of lipomatous muscle alterations and the efficacy of long-term enzyme replacement therapy.Genotype-phenotype correlation in Pompe disease, a step forward.Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.Autophagy in Natural History and After ERT in Glycogenosis Type II.Familial Pompe DiseaseQuality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up.Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational studyTherapeutic advances in the management of Pompe disease and other metabolic myopathiesFamilial adult-onset Pompe disease associated with unusual clinical and histological features.Recent advances in clinical neurogenetics.Extended phenotype description and new molecular findings in late onset glycogen storage disease type II: a northern Italy population study and review of the literature.Ten years of the international Pompe survey: patient reported outcomes as a reliable tool for studying treated and untreated children and adults with non-classic Pompe disease.Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel.Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis.European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience.Clinical and molecular aspects of 30 patients with late-onset Pompe disease (LOPD): unusual features and response to treatment.Clinical Analysis of Algerian Patients with Pompe Disease.Enzyme replacement in neuronal storage disorders in the pediatric population.The brain in late-onset glycogenosis II: a structural and functional MRI study.[Diagnosis and therapy of late onset Pompe disease].S3.1 Are there ERT defined guidelinesfor Pompe disease?S2.3 Therapeutic approaches in the late onsetform of GSD II.Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD).Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease.Cessation and resuming of alglucosidase alfa in Pompe disease: a retrospective analysis.Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study.Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients.Vacuolated PAS-Positive Lymphocytes on Blood Smear: An Easy Screening Tool and a Possible Biomarker for Monitoring Therapeutic Responses in Late Onset Pompe Disease (LOPD)Orthopedic management of patients with Pompe disease: a retrospective case series of 8 patients
P2860
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P2860
Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years.
description
2011 nî lūn-bûn
@nan
2011年の論文
@ja
2011年学术文章
@wuu
2011年学术文章
@zh
2011年学术文章
@zh-cn
2011年学术文章
@zh-hans
2011年学术文章
@zh-my
2011年学术文章
@zh-sg
2011年學術文章
@yue
2011年學術文章
@zh-hant
name
Observational clinical study i ...... ent therapy for up to 4 years.
@en
Observational clinical study i ...... ent therapy for up to 4 years.
@nl
type
label
Observational clinical study i ...... ent therapy for up to 4 years.
@en
Observational clinical study i ...... ent therapy for up to 4 years.
@nl
prefLabel
Observational clinical study i ...... ent therapy for up to 4 years.
@en
Observational clinical study i ...... ent therapy for up to 4 years.
@nl
P2093
P2860
P50
P1433
P1476
Observational clinical study i ...... ment therapy for up to 4 years
@en
P2093
E Pegoraro
G Crescimanno
G Di Iorio
Italian GSDII Group
P2860
P2888
P304
P356
10.1007/S00415-011-6293-5
P577
2011-11-12T00:00:00Z