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October 1, 2004Archives of Neurology

Phenotypic Features and Genetic Findings in 2 Chinese Families With Miyoshi Distal Myopathy

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Key result

Genetic analysis of 4 patients with Miyoshi distal myopathy from 2 Chinese families revealed compound heterozygous mutations in the dysferlin gene, including 3 novel mutations.

Population

4 patients with Miyoshi distal myopathy from 2 unrelated Chinese families

Design

Case_series

Authors

LRLong-Sun RoGLGuey‐Jen Lee‐ChenTLTzu-Ching Lin

Discussion

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Member takes

Overview

May aid genetic diagnosis in similar families; leaves open validation and prevalence studies in larger cohorts.

Study Design

Type

Case Report (n=4)

Structured PICO

P
Population
4 patients with Miyoshi distal myopathy from 2 unrelated Chinese families.
O
Outcome
Clinical, neurophysiological, histopathological, and genetic features

Identified novel compound heterozygous mutations in the dysferlin gene in Chinese families with Miyoshi distal myopathy.

Cite This Study

Ro et al. (2004) conducted a case report in Miyoshi distal myopathy (n=4). Genetic analysis and clinical observation was evaluated on Clinical, neurophysiological, histopathological, and genetic features. Genetic analysis of 4 patients with Miyoshi distal myopathy from 2 Chinese families revealed compound heterozygous mutations in the dysferlin gene, including 3 novel mutations.

synapsesocial.com/papers/6a95c415fbedde85bb420ed8https://doi.org/10.1001/archneur.61.10.1594
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Also Consider

Synapse has enriched 3 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Genomic organization of the dysferlin gene and novel mutations in Miyoshi myopathy2001 · 146 citations
  2. 2Linkage of Miyoshi myopathy (distal autosomal recessive muscular dystrophy) locus to chromosome 2p12-141995 · 147 citations
  3. 3Dysferlin is a Plasma Membrane Protein and is Expressed Early in Human Development1999 · 297 citations