spinal muscular atrophy with congenital bone fractures 1

Rare Disease

MONDO:0014806

Also known as:

SMABF1TRIP4 prenatal-onset spinal muscular atrophy with congenital bone fracturesprenatal-onset spinal muscular atrophy with congenital bone fractures caused by mutation in TRIP4

Any prenatal-onset spinal muscular atrophy with congenital bone fractures in which the cause of the disease is a mutation in the TRIP4 gene.

Genes

Clinical Trials

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Newborn Screening

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Patient Groups

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Patient-Friendly Information

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Companies Active in spinal muscular atrophy with congenital bone fractures 1

3 approved

Organizations with approved therapies for this disease

Novartis Gene Therapies

Other

Approved

Biogen Inc.

Other

Approved

Genentech, Inc.

Other

Approved

View all companies active in spinal muscular atrophy with congenital bone fractures 1

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