radioulnar synostosis with amegakaryocytic thrombocytopenia 1

Rare Disease

MONDO:0024558

Also known as:

HOXA11 radio-ulnar synostosis-amegakaryocytic thrombocytopenia syndromeradio-ulnar synostosis-amegakaryocytic thrombocytopenia syndrome caused by mutation in HOXA11radioulnar synostosis with amegakaryocytic thrombocytopenia 1

Any radio-ulnar synostosis-amegakaryocytic thrombocytopenia syndrome in which the cause of the disease is a mutation in the HOXA11 gene.

Genes

Clinical Trials

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Newborn Screening

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Patient Groups

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Companies Active in radioulnar synostosis with amegakaryocytic thrombocytopenia 1

3 companies

Organizations with orphan designations or approved therapies for this disease

Amgen Inc

Other

Designated

Rigel Pharmaceuticals, Inc.

Other

Designated

Sobi, Inc.

Other

Designated

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