Natural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpoints.

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Natural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpoints. is …
instance of (P31):
scholarly articleQ13442814

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P356DOI10.1002/ACN3.774
P932PMC publication ID6562036
P698PubMed publication ID31211167

P50authorMichael HannaQ20876911
John S ThorntonQ57050521
Tarek A YousryQ58364812
Jean-Yves HogrelQ58818465
Volker StraubQ59878147
Julia R DahlqvistQ85559719
Anna G MayhewQ90185542
Pierre G CarlierQ90591135
Kieren G HollingsworthQ91380826
Alexander Peter MurphyQ92808178
P2093author name stringTanya Stojkovic
Christopher D J Sinclair
John Vissing
Michelle Eagle
Jasper Morrow
Laurence E Lee
Meredith K James
Stephen Wastling
Tracey A Willis
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Upper Limb Evaluation in Duchenne Muscular Dystrophy: Fat-Water Quantification by MRI, Muscle Force and Function Define Endpoints for Clinical TrialsQ31131975
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Three-point Dixon technique for true water/fat decomposition with B0 inhomogeneity correctionQ33577161
Prevalence of genetic muscle disease in Northern England: in-depth analysis of a muscle clinic populationQ33685609
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Flow cytometry for the analysis of α-dystroglycan glycosylation in fibroblasts from patients with dystroglycanopathiesQ34873693
Quantitative muscle MRI as an assessment tool for monitoring disease progression in LGMD2I: a multicentre longitudinal studyQ34963315
FKRP (826C>A) frequently causes limb-girdle muscular dystrophy in German patientsQ35445374
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Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort.Q37114363
The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter studyQ37298079
Reliability of the North Star Ambulatory Assessment in a multicentric setting.Q37530045
Upper arm and cardiac magnetic resonance imaging in Duchenne muscular dystrophyQ37577885
Quantitative magnetic resonance imaging in limb-girdle muscular dystrophy 2I: a multinational cross-sectional studyQ37610370
Longitudinal functional and NMR assessment of upper limbs in Duchenne muscular dystrophy.Q39213123
Quantifying fat replacement of muscle by quantitative MRI in muscular dystrophy.Q39409381
B4GALNT2 (GALGT2) Gene Therapy Reduces Skeletal Muscle Pathology in the FKRP P448L Mouse Model of Limb Girdle Muscular Dystrophy 2I.Q39453819
Muscle and heart function restoration in a limb girdle muscular dystrophy 2I (LGMD2I) mouse model by systemic FKRP gene deliveryQ40610977
The phenotype of limb-girdle muscular dystrophy type 2IQ44411539
Subepicardial dysfunction leads to global left ventricular systolic impairment in patients with limb girdle muscular dystrophy 2I.Q45384428
The quality of life in genetic neuromuscular disease questionnaire: Rasch validation of the French versionQ47579115
216th ENMC international workshop: Clinical readiness in FKRP related myopathies January 15-17, 2016 Naarden, The NetherlandsQ48793388
The 6-minute walk test, motor function measure and quantitative thigh muscle MRI in Becker muscular dystrophy: A cross-sectional study.Q50858360
Contractile properties are disrupted in Becker muscular dystrophy, but not in limb girdle type 2I.Q52673776
Significant response to immune therapies in a case of subacute necrotizing myopathy and FKRP mutations.Q53347402
Prevalence, mutation spectrum and phenotypic variability in Norwegian patients with Limb Girdle Muscular Dystrophy 2IQ59697187
The fatigue severity scale. Application to patients with multiple sclerosis and systemic lupus erythematosusQ69772148
ACTIVLIM: a Rasch-built measure of activity limitations in children and adults with neuromuscular disordersQ80157260
Inflammation and response to steroid treatment in limb-girdle muscular dystrophy 2IQ80186224
Three-point technique of fat quantification of muscle tissue as a marker of disease progression in Duchenne muscular dystrophy: preliminary studyQ80235144
Quantitative MRI and loss of free ambulation in Duchenne muscular dystrophyQ85398934
Magnetic resonance imaging in Duchenne muscular dystrophy: longitudinal assessment of natural history over 18 monthsQ86661606
Quantitative MRI and strength measurements in the assessment of muscle quality in Duchenne muscular dystrophyQ87419552
229th ENMC international workshop: Limb girdle muscular dystrophies - Nomenclature and reformed classification Naarden, the Netherlands, 17-19 March 2017Q90589536
P433issue6
P921main subjectmuscular dystrophyQ1137767
P304page(s)1033-1045
P577publication date2019-05-16
P1433published inAnnals of clinical and translational neurologyQ27725312
P1476titleNatural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpoints
P478volume6

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cites work (P2860)
Q90022892Follow-up of late-onset Pompe disease patients with muscle magnetic resonance imaging reveals increase in fat replacement in skeletal muscles
Q92234239Natural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpoints

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