myofibrillar myopathy 2

Rare Disease

MONDO:0012130

Also known as:

CRYAB autosomal dominant distal myopathyalpha-B crystallinopathyautosomal dominant distal myopathy caused by mutation in CRYAB

Any autosomal dominant distal myopathy in which the cause of the disease is a mutation in the CRYAB gene.

Genes

Clinical Trials

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Newborn Screening

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Patient Groups

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Companies Active in myofibrillar myopathy 2

2 companies

Organizations with orphan designations or approved therapies for this disease

BridgeBio Pharma, Inc.

Other

Designated

FoldRx Pharmaceuticals, Inc., a subsidiary of Pfizer Inc.

Other

Designated

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