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Article

Early clinical and pre-clinical therapy development in Nemaline myopathy.

Expert opinion on therapeutic targets - 1 Oct 2022

Fisher Gemma, Mackels Laurane, Markati Theodora, Sarkozy Anna, Ochala Julien, Jungbluth Heinz, Ramdas Sithara, Servais Laurent

Abstract excerpt

INTRODUCTION: Nemaline myopathies (NM) represent a group of clinically and genetically heterogeneous congenital muscle disorders with the common denominator of nemaline rods on muscle biopsy. NEB and ACTA1 are the most common causative genes. Currently, available treatments are supportive. AREAS COVERED: We explored experimental treatments for NM, identifying at least eleven mainly pre-clinical approaches...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Identifiers

PMID: 36524401
DOI: 10.1080/14728222.2022.2157258

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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