Typical nemaline myopathy

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Typical nemaline myopathy is a moderate neonatal form of nemaline myopathy (NM; see this term) characterized by facial and skeletal muscle weakness and mild respiratory involvement.

Data from Orphanet are used to provide information on a disease's name, synonym(s), and overview. Reference: Access aggregated data from Orphanet at Orphadata. Orphadata: Free access data from Orphanet. © INSERM 1999. Available on http://www.orphadata.org. Data version June 2024

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Typical nemaline myopathy?

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Advocacy Organizations

A Foundation Building Strength

A Foundation Building Strength’s mission is to find treatments for Nemaline Myopathy (NM). AFBS works to address issues for our NM families from all angles, including advancing research, providing information about care, offering family support groups and ways to connect, and creating opportunities for in-person interaction and education at conferences.

Project CASK

VISION Project CASK believes in a world free of the life limitations and debilitating effects of CASK gene disorders. ​MISSION To accelerate breakthroughs in research to develop treatments and a cure for CASK gene disorders. ​VALUES Collaboration. Transparency. Urgency.

Team Titin

Team Titin, Inc.'s mission is to serve those living with, caring for, or researching titin (TTN) related muscle and heart disorders. To achieve this mission, we focus on four key pillars: Connection, Advocacy, Care, and Research.

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Clinical Trials

For a list of clinical trials in this disease area, please click here.