Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
about
The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?Practical Recommendations for Diagnosis and Management of Respiratory Muscle Weakness in Late-Onset Pompe DiseaseMuscle 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.Late-Onset Glycogen Storage Disease Type II (Pompe's Disease) with a Novel Mutation: A Malaysian Experience.Urge incontinence and gastrointestinal symptoms in adult patients with pompe disease: a cross-sectional surveyGenotype-phenotype correlation in Pompe disease, a step forward.B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case StudyPompe disease: literature review and case seriesComparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe miceDiagnosis and treatment of late-onset Pompe disease in the Middle East and North Africa region: consensus recommendations from an expert groupSafety 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 programStimulation of Respiratory Motor Output and Ventilation in a Murine Model of Pompe Disease by Ampakines.A conceptual disease model for adult Pompe disease.Muscle MRI Findings in Childhood/Adult Onset Pompe Disease Correlate with Muscle Function.Management of a pregnancy complicated by pompe disease.Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up.Prevalence of Pompe disease in 3,076 patients with hyperCKemia and limb-girdle muscular weakness.Therapeutic advances in the management of Pompe disease and other metabolic myopathiesDistinct disease phenotypes linked to different combinations of GAA mutations in a large late-onset GSDII sibshipAdult-onset Pompe's disease presenting with insidious hypercapnic respiratory failure.Targeting Wolman Disease and Cholesteryl Ester Storage Disease: Disease Pathogenesis and Therapeutic Development.The respiratory neuromuscular system in Pompe disease.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.Ethical considerations of population screening for late-onset genetic disease.Axons to Exons: the Molecular Diagnosis of Rare Neurological Diseases by Next-Generation Sequencing.Guidelines for the diagnosis, treatment and clinical monitoring of patients with juvenile and adult 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.Hereditary myopathies with early respiratory insufficiency in adults.European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience.The investigation and management of metabolic myopathies.From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides.Clinical and molecular aspects of 30 patients with late-onset Pompe disease (LOPD): unusual features and response to treatment.The influence of a polymorphism in the gene encoding angiotensin converting enzyme (ACE) on treatment outcomes in late-onset Pompe patients receiving alglucosidase alfaEarly is better? A new algorithm for early diagnosis in late onset Pompe disease (LOPD).Effects of immune modulation therapy in the first Croatian infant diagnosed with Pompe disease: a 3-year follow-up study.Should patients with asymptomatic pompe disease be treated? A nationwide study in France.[Diagnosis and therapy of late onset Pompe disease].Protein Complexation and pH Dependent Release Using Boronic Acid Containing PEG-Polypeptide Copolymers.Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in children affected by Pompe Disease.
P2860
Q27021472-167FD85D-70C3-4050-962C-9EF728928002Q28067387-FA13E597-5A72-49D5-887B-8ED72474ABAAQ31052050-16AEEAE8-DDC4-460A-9C2D-A8CB77242E94Q33908205-7D8DDA58-3429-4826-98BE-58AF45E5513CQ34560232-1731A18C-161F-4C0C-824F-6355B1B90B22Q34598948-6EE67DF0-3E7E-4593-9D24-B48A6C494188Q34763584-3E1C650A-5F44-4DBC-9A9B-02AA654DB3B6Q35029971-458C1574-9A25-475E-A303-014CA4332813Q35651103-862DE697-338C-445D-BFAB-E238DF3ED75AQ35809528-A4431D9B-E12F-4548-AA73-C273F38A2AD3Q35863802-8258F440-2E52-4267-903D-A4D6B03D65A8Q36049311-A824C7F6-B69A-4141-803F-0AA20E26B986Q36061849-276BC382-9915-4588-BD90-CD8EE55D117FQ36154874-5B64650A-C9A5-4609-B742-9F2B5B28A1D7Q36470470-F91977AF-D6C8-418E-B732-F7E5827ABAD3Q36582225-B8C34563-9F67-4971-9EDF-7A256A5BFADBQ37113363-14F609E8-7C27-4DF3-A4C2-60F9C752555BQ37127561-B04507DA-6B9C-4CDD-BC78-E293C356ABB7Q37365514-CA7F9F6A-5D0F-411B-BBF8-A802701605C0Q37600615-7EE7640B-EF65-4A79-B6BB-EA0E84E6E4DFQ37717441-24F7FC16-FD9D-4B2C-A45C-4DB455082C9BQ38116744-8F792160-1535-416D-8BCB-F18F1BB24158Q38238918-78418B02-71B8-4CEE-AB27-380F41D01C91Q38354133-B559580E-B1BE-42A2-99AA-70099D99F224Q38569822-5478F657-BD33-464D-B7D5-9FE20275F4A6Q38675468-B8132A9C-AEFB-4010-BFBE-02750F6D1855Q38800985-907BD962-5348-450A-BF06-C2408D63C9E8Q38884347-3575D7B7-EF9A-4698-9383-E840D556D952Q38970452-ED5DC0AC-D20A-43FB-9954-D0F35A62472AQ39287288-4CDFD7D9-8036-451F-85F1-29D83E3CF6A1Q41330451-3671C2B6-B6B0-471F-8B0E-A7114AA60ED3Q41387647-D2713CE4-666E-49FD-9B62-C4DFB2DAE335Q41448556-09953CAD-2361-418B-996B-AE93A3BB5866Q42403394-33578541-D8B3-4D93-83ED-EE0A79AACE0CQ42787786-2DB76360-A6F7-41F5-9FF3-72F6094C26D4Q43418166-6CFE1D88-4833-40AC-B1F1-6DB1566BA56EQ44725647-E432E13F-F012-4327-AC9D-A5015AD67475Q44767256-9107564A-C895-4909-9E9B-7095BB21EE2FQ44805646-9F1EAE4C-5903-4464-888B-B8D61115DF29Q45324511-99EF0EC9-3729-4CFC-8051-0D1B16A7E2DA
P2860
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
description
2012 nî lūn-bûn
@nan
2012 թուականի Օգոստոսին հրատարակուած գիտական յօդուած
@hyw
2012 թվականի օգոստոսին հրատարակված գիտական հոդված
@hy
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
name
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
@ast
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
@en
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
@nl
type
label
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
@ast
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
@en
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
@nl
prefLabel
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
@ast
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
@en
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
@nl
P2860
P1433
P1476
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
@en
P2093
Antonio Toscano
Benedikt Schoser
P2860
P2888
P304
P356
10.1007/S00415-012-6636-X
P577
2012-08-28T00:00:00Z
P5875
P6179
1041415385