Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease.

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Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease. is …
instance of (P31):
scholarly articleQ13442814

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P356DOI10.1007/S00415-013-6980-5
P698PubMed publication ID23749294

P50authorMarianne RohrbachQ51236258
Thomas HundsbergerQ56541747
P2093author name stringKai M Rösler
Lukas Kern
P2860cites workEnzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial.Q42629452
36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapyQ42634296
Diagnostic efficacy of the fluorometric determination of enzyme activity for Pompe disease from dried blood specimens compared with lymphocytes-possibility for newborn screeningQ43208556
Diagnostic criteria for late-onset (childhood and adult) Pompe diseaseQ43802882
Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease.Q44376591
Orphan drugs for rare diseases: is it time to revisit their special market access status?Q44871497
Clinical trials of orphan medicinesQ45324849
Pompe disease: from new views on pathophysiology to innovative therapeutic strategiesQ45866303
Disease severity in children and adults with Pompe disease related to age and disease durationQ47772980
Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trialQ47869064
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
Making diagnosis of Pompe disease at a presymptomatic stage: to treat or not to treat?Q54519754
Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type IIQ64042342
Cerebral vascular accidents in patients over the age of 60. I. General considerationsQ74508594
Carrier frequency for glycogen storage disease type II in New York and estimates of affected individuals born with the diseaseQ77304104
Cognitive and adaptive functioning of children with infantile Pompe disease treated with enzyme replacement therapy: long-term follow-upQ83264951
First experience with enzyme replacement therapy during pregnancy and lactation in Pompe diseaseQ85034949
Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational studyQ21202855
Autophagy and mitochondria in Pompe disease: nothing is so new as what has long been forgottenQ27015911
Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapyQ28586585
Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe diseaseQ30489156
Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trialQ33941289
A randomized study of alglucosidase alfa in late-onset Pompe's diseaseQ34110220
Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patientsQ34554310
Pompe disease diagnosis and management guideline.Q34567528
Screening for Pompe disease using a rapid dried blood spot method: experience of a clinical diagnostic laboratoryQ34608980
Lysosomal storage diseases: diagnostic confirmation and management of presymptomatic individualsQ34628634
Consensus treatment recommendations for late-onset Pompe diseaseQ34634354
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature reviewQ34641771
Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe diseaseQ35087282
Methods of formal consensus in classification/diagnostic criteria and guideline developmentQ35091202
Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapyQ35102705
The natural course of non-classic Pompe's disease; a review of 225 published casesQ36245480
Methods for a prompt and reliable laboratory diagnosis of Pompe disease: report from an international consensus meetingQ37031665
Pompe disease: a review of the current diagnosis and treatment recommendations in the era of enzyme replacement therapyQ37182561
Pompe's diseaseQ37302098
Therapeutic approaches in glycogen storage disease type II/Pompe Disease.Q37329535
Management and treatment of glycogenosis type II.Q37338760
Diagnosis of glycogenosis type II.Q37338764
The Brazilian consensus on the management of Pompe disease.Q37600014
Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe diseaseQ38356012
P433issue9
P921main subjectenzyme replacement therapyQ916766
P304page(s)2279-2285
P577publication date2013-06-08
P1433published inJournal of NeurologyQ6295649
P1476titleSwiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease.
P478volume260

Reverse relations

cites work (P2860)
Q30398761Can stapedius reflex testing objectively measure muscle function in Pompe patients?
Q51717385Cessation and resuming of alglucosidase alfa in Pompe disease: a retrospective analysis.
Q39287288European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience.
Q34727909Objective consensus from decision trees
Q38800985Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel
Q47404438Pompe disease in Austria: clinical, genetic and epidemiological aspects
Q35029971Pompe disease: literature review and case series
Q34151422The European Gaucher Alliance: a survey of member patient organisations' activities, healthcare environments and concerns

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