نتایج جستجو برای: van laere syndrome

تعداد نتایج: 686720  

Journal: :Rheumatology 2021

Abstract Background/Aims Myopathies due to inborn errors of metabolism can be difficult differentiate from inflammatory myopathies. Careful history, examination and laboratory tests are required establish the diagnosis. We present a case Riboflavin Transport Deficiency (Brown-Vialetto-Van Laere syndrome) masquerading as an myopathy. Methods A 37-year-old lady presented with severe proximal musc...

Journal: :Neurobiology of disease 2012
Paloma González-Pérez Yubing Lu Ru-Ju Chian Peter C Sapp Rudolph E Tanzi Lars Bertram Diane McKenna-Yasek Fen-Biao Gao Robert H Brown

UNLABELLED Genetic variants in UBQLN1 gene have been linked to neurodegeneration and mutations in UBQLN2 have recently been identified as a rare cause of amyotrophic lateral sclerosis (ALS). OBJECTIVE To test if genetic variants in UBQLN1 are involved in ALS. METHODS 102 and 94 unrelated patients with familial and sporadic forms of ALS were screened for UBQLN1 gene mutations. Single nucleot...

2015
Slavé Petrovski Vandana Shashi Steven Petrou Kelly Schoch Keisha Melodi McSweeney Ryan S. Dhindsa Brian Krueger Rebecca Crimian Laura E. Case Roha Khalid Maysantoine A. El-Dairi Yong-Hui Jiang Mohamad A. Mikati David B. Goldstein

Genetically targeted therapies for rare Mendelian conditions are improving patient outcomes. Here, we present the case of a 20-mo-old female suffering from a rapidly progressing neurological disorder. Although diagnosed initially with a possible autoimmune condition, analysis of the child's exome resulted in a diagnosis of Brown-Vialetto-Van Laere syndrome 2 (BVVLS2). This new diagnosis led to ...

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