scholarly article | Q13442814 |
P356 | DOI | 10.1007/S00415-013-6980-5 |
P698 | PubMed publication ID | 23749294 |
P50 | author | Marianne Rohrbach | Q51236258 |
Thomas Hundsberger | Q56541747 | ||
P2093 | author name string | Kai M Rösler | |
Lukas Kern | |||
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Orphan drugs for rare diseases: is it time to revisit their special market access status? | Q44871497 | ||
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Pompe disease: from new views on pathophysiology to innovative therapeutic strategies | Q45866303 | ||
Disease severity in children and adults with Pompe disease related to age and disease duration | Q47772980 | ||
Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial | Q47869064 | ||
Enzyme replacement therapy in adult-onset glycogenosis II: is quantitative muscle MRI helpful? | Q50781892 | ||
Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years. | Q50790826 | ||
Changes in skeletal muscle qualities during enzyme replacement therapy in late-onset type II glycogenosis: temporal and spatial pattern of mass vs. strength response. | Q51771787 | ||
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Cerebral vascular accidents in patients over the age of 60. I. General considerations | Q74508594 | ||
Carrier frequency for glycogen storage disease type II in New York and estimates of affected individuals born with the disease | Q77304104 | ||
Cognitive and adaptive functioning of children with infantile Pompe disease treated with enzyme replacement therapy: long-term follow-up | Q83264951 | ||
First experience with enzyme replacement therapy during pregnancy and lactation in Pompe disease | Q85034949 | ||
Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational study | Q21202855 | ||
Autophagy and mitochondria in Pompe disease: nothing is so new as what has long been forgotten | Q27015911 | ||
Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapy | Q28586585 | ||
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Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial | Q33941289 | ||
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Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease | Q35087282 | ||
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Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy | Q35102705 | ||
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Methods for a prompt and reliable laboratory diagnosis of Pompe disease: report from an international consensus meeting | Q37031665 | ||
Pompe disease: a review of the current diagnosis and treatment recommendations in the era of enzyme replacement therapy | Q37182561 | ||
Pompe's disease | Q37302098 | ||
Therapeutic approaches in glycogen storage disease type II/Pompe Disease. | Q37329535 | ||
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Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease | Q38356012 | ||
P433 | issue | 9 | |
P921 | main subject | enzyme replacement therapy | Q916766 |
P304 | page(s) | 2279-2285 | |
P577 | publication date | 2013-06-08 | |
P1433 | published in | Journal of Neurology | Q6295649 |
P1476 | title | Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease. | |
P478 | volume | 260 |
Q30398761 | Can stapedius reflex testing objectively measure muscle function in Pompe patients? |
Q51717385 | Cessation and resuming of alglucosidase alfa in Pompe disease: a retrospective analysis. |
Q39287288 | European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience. |
Q34727909 | Objective consensus from decision trees |
Q38800985 | Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel |
Q47404438 | Pompe disease in Austria: clinical, genetic and epidemiological aspects |
Q35029971 | Pompe disease: literature review and case series |
Q34151422 | The European Gaucher Alliance: a survey of member patient organisations' activities, healthcare environments and concerns |
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