Departament of Neurology, Escola Paulista de Medicina, Universidade Federal de São Paulo, São Paulo, Brazil.
Departament of Neurology, Escola Paulista de Medicina, Universidade Federal de São Paulo, São Paulo, Brazil.
Clin Neurol Neurosurg. 2024 Apr;239:108213. doi: 10.1016/j.clineuro.2024.108213. Epub 2024 Feb 28.
Hereditary motor and sensory neuropathy with proximal dominant involvement (HMSN-P), or, Okinawa type, is a rare neuromuscular disorder characterized by proximal dominant neurogenic atrophy and distal sensory alterations with an autosomal dominant inheritance pattern. We present a case of a Brazilian woman of Okinawan ancestry, with symmetrical proximal weakness, fasciculations, absent patellar reflexes and positive familial history for the same symptoms. These findings led to genetic testing, which identified a variant in the TFG gene (c.854 C>T;p.(Pro285Leu), confirming the diagnosis of HMSN-P. HMSN-P seemed to be restricted to populations in Okinawa, however, other HMSN-P cases were described in several parts of the world, especially in South America. This case report emphasizes the importance of considering HMSN-P in patients presenting with clinical features resembling proximal myopathy, especially in individuals with Okinawan ancestry.
遗传性运动感觉神经病伴近端优势受累(HMSN-P),又称冲绳型,是一种罕见的神经肌肉疾病,其特征为近端优势神经源性萎缩和远端感觉改变,呈常染色体显性遗传模式。我们报告了一例巴西裔冲绳女性患者,表现为对称性近端肌无力、肌束震颤、髌反射消失,并具有相同症状的阳性家族史。这些发现促使进行基因检测,结果发现 TFG 基因的变异(c.854C>T;p.(Pro285Leu)),从而确诊为 HMSN-P。HMSN-P 似乎仅限于冲绳人群,但在世界其他地区,包括南美洲,也有其他 HMSN-P 病例的报道。本病例报告强调了在具有类似近端肌病临床特征的患者中,考虑 HMSN-P 的重要性,尤其是在具有冲绳裔背景的个体中。