spinal muscular atrophy with congenital bone fractures 2

Rare Disease

MONDO:0014807

Also known as:

ASCC1 prenatal-onset spinal muscular atrophy with congenital bone fracturesSMABF2prenatal-onset spinal muscular atrophy with congenital bone fractures caused by mutation in ASCC1

Any prenatal-onset spinal muscular atrophy with congenital bone fractures in which the cause of the disease is a mutation in the ASCC1 gene.

Genes

Clinical Trials

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Newborn Screening

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Patient Groups

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Companies Active in spinal muscular atrophy with congenital bone fractures 2

3 companies

Organizations with orphan designations or approved therapies for this disease

Biogen Inc.

Other

Approved

Genentech, Inc.

Other

Approved

Novartis Gene Therapies

Other

Approved

View all companies