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Neuromuscular Disorders : NMD
|
September 5, 2015
Novel mutations in DNAJB6 gene cause a very severe early-onset limb-girdle muscular dystrophy 1D disease
Johanna Palmio, Per Harald Jonson, Anni Evilä, et al.
Neuromuscular Disorders : NMD
|
July 8, 2010
Inheritance patterns and phenotypic features of myofibrillar myopathy associated with a BAG3 mutation
Zagaa Odgerel, Anna Sarkozy, Hee-Suk Lee, et al.
Journal of Medical Internet Research
|
May 30, 2025
The Potential to Leverage Real-World Data for Pediatric Clinical Trials: A Proof-of-Concept Study
Jens Declerck, Joanne Lee, Anando Sen, et al.
Neuromuscular Disorders : NMD
|
December 28, 2021
Patient reported quality of life in limb girdle muscular dystrophy
Laurel V Kovalchick, Kameron Bates, Jeffrey Statland, et al.
European Journal of Human Genetics : EJHG
|
June 2, 2011
Phenotypic heterogeneity in British patients with a founder mutation in the FHL1 gene
Anna Sarkozy, Christian Windpassinger, Judith Hudson, et al.
Neuromuscular Disorders : NMD
|
July 19, 2017
A 'second truncation' in TTN causes early onset recessive muscular dystrophy
Elizabeth Harris, Ana Töpf, Anna Vihola, et al.
Neuromuscular Disorders : NMD
|
April 10, 2023
A pathogenic CTBP1 variant featuring HADDTS with dystrophic myopathology
Hazim Kadhim, Eliane El-Howayek, Sandra Coppens, et al.
Endocrine Connections
|
September 28, 2023
Is ongoing testosterone required after pubertal induction in Duchenne muscular dystrophy?
Claire L Wood, Kieren G Hollingsworth, Edrina Bokaie, et al.
Disease Models & Mechanisms
|
February 19, 2020
Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)
Raffaella Willmann, Joanne Lee, Cathy Turner, et al.
European Journal of Medical Genetics
|
January 13, 2023
Expanding the neurodevelopmental phenotype associated with HK1 de novo heterozygous missense variants
Rebecca L Poole, Mihaly Badonyi, Alison Cozens, et al.
Page
of 38
Search research articles
Search
Showing results (121-130 of 374) with videos related to
Sort By:
Page
of 38
Neuromuscular Disorders : NMD
|
September 5, 2015
Novel mutations in DNAJB6 gene cause a very severe early-onset limb-girdle muscular dystrophy 1D disease
Johanna Palmio, Per Harald Jonson, Anni Evilä, et al.
Neuromuscular Disorders : NMD
|
July 8, 2010
Inheritance patterns and phenotypic features of myofibrillar myopathy associated with a BAG3 mutation
Zagaa Odgerel, Anna Sarkozy, Hee-Suk Lee, et al.
Journal of Medical Internet Research
|
May 30, 2025
The Potential to Leverage Real-World Data for Pediatric Clinical Trials: A Proof-of-Concept Study
Jens Declerck, Joanne Lee, Anando Sen, et al.
Neuromuscular Disorders : NMD
|
December 28, 2021
Patient reported quality of life in limb girdle muscular dystrophy
Laurel V Kovalchick, Kameron Bates, Jeffrey Statland, et al.
European Journal of Human Genetics : EJHG
|
June 2, 2011
Phenotypic heterogeneity in British patients with a founder mutation in the FHL1 gene
Anna Sarkozy, Christian Windpassinger, Judith Hudson, et al.
Neuromuscular Disorders : NMD
|
July 19, 2017
A 'second truncation' in TTN causes early onset recessive muscular dystrophy
Elizabeth Harris, Ana Töpf, Anna Vihola, et al.
Neuromuscular Disorders : NMD
|
April 10, 2023
A pathogenic CTBP1 variant featuring HADDTS with dystrophic myopathology
Hazim Kadhim, Eliane El-Howayek, Sandra Coppens, et al.
Endocrine Connections
|
September 28, 2023
Is ongoing testosterone required after pubertal induction in Duchenne muscular dystrophy?
Claire L Wood, Kieren G Hollingsworth, Edrina Bokaie, et al.
Disease Models & Mechanisms
|
February 19, 2020
Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)
Raffaella Willmann, Joanne Lee, Cathy Turner, et al.
European Journal of Medical Genetics
|
January 13, 2023
Expanding the neurodevelopmental phenotype associated with HK1 de novo heterozygous missense variants
Rebecca L Poole, Mihaly Badonyi, Alison Cozens, et al.
Page
of 38