• Source: CHAMP1-associated intellectual disability syndrome
  • CHAMP1-associated intellectual disability syndrome, also known as autosomal dominant intellectual disability type 40, is a rare genetic disorder characterized by intellectual disabilities, developmental delays, facial dysmorphisms, and other anomalies.


    Signs and symptoms


    Individuals with the disorder often show the following signs and symptoms:

    Intellectual disabilities
    Speech delays
    Epicanthic fold
    Drooping of the lower lip
    Feeding difficulties
    Hypotonia of the face
    Ataxia
    Hyperopia
    High palate
    Widespread developmental delays
    Gastroesophageal reflux
    Widespread hypotonia
    Decreased sense of pain
    Joint hypermobility
    Long face
    Constantly open mouth
    Prognathism (pointy chin)
    High occurrence of respiratory tract infections
    Short philtrum
    Stereotypy
    Tented upper lip vermilion
    Upslanted palpebral fissures
    Strabismus
    Autism/autistic-like behaviour


    Causes


    As its name suggests, the condition is caused by mutations in the CHAMP1 gene, in chromosome 13q34. These mutations are most often missense or nonsense mutations. They are usually sporadic, meaning the condition is not inherited from the parents. However, if people with the disorder were to reproduce, they would have a 1 in 2 chance of giving their children a copy of the gene, because the disorder is autosomal dominant, which means that only one copy of a mutated gene (whether inherited or from a spontaneous error in cell division) is needed to pass it on to a child.


    Epidemiology


    According to OMIM, only 36 cases have been described in medical literature.
    Worldwide there are only around 170 known cases as confirmed by the CHAMP1 non-profits CHAMP1 UK & The CHAMP1 Research Foundation.


    Support for those affected


    There are 2 main charities/non-profits offering support for those affected by CHAMP1:
    CHAMP1 UK &
    The CHAMP1 Research Foundation


    References

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