Practice of sports during childhood or adolescence correlates with an earlier onset and more rapidly progressing phenotype in dysferlinopathies. To determine if this correlation relates to greater mus

NIAID Data Ecosystem20

Population characteristics subdivided by brain dystrophin involvement and corticosteroids.

神经肌肉疾病临床研究

dystrophin蛋白影响分析

Population characteristics subdivided by brain dystrophin involvement and corticosteroids.

NIAID Data Ecosystem20

UK Facioscapulohumeral Muscular Dystrophy (FSHD) Patient Registry

罕见病患者登记

神经肌肉疾病临床研究

The UK FSHD Patient Registry aims to recruit any individual, from anywhere within the United Kingdom, with a diagnosis of fshd who may be interested in becoming involved in future planned clinical tri

DataCite Commons2021-06-14 更新40

Sporadic late-onset nemaline myopathy: clinical spectrum, survival and treatment outcomes

神经肌肉疾病临床研究

罕见病治疗疗效评估

OBJECTIVES: To describe the clinical phenotype, long-term treatment outcome and overall survival of sporadic late onset nemaline myopathy (SLONM) with or without a monoclonal protein (MP). METHODS: we

NIAID Data Ecosystem20

Characteristics of the 120 patients enrolled for the SfEMG study.

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