U.S. flag

An official website of the United States government

Format

Send to:

Choose Destination

Aase-Smith syndrome

MedGen UID:
66316
Concept ID:
C0220686
Disease or Syndrome
Synonyms: AASE-SMITH SYNDROME I; Hydrocephalus cleft palate joint contractures; Joint contractures with other abnormalities
SNOMED CT: Hydrocephalus with cleft palate and joint contracture syndrome (718576001); Aase Smith type 1 syndrome (718576001); Aase Smith I syndrome (718576001)
Modes of inheritance:
Autosomal dominant inheritance
MedGen UID:
141047
Concept ID:
C0443147
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in heterozygotes. In the context of medical genetics, an autosomal dominant disorder is caused when a single copy of the mutant allele is present. Males and females are affected equally, and can both transmit the disorder with a risk of 50% for each child of inheriting the mutant allele.
 
Monarch Initiative: MONDO:0007839
OMIM®: 147800
Orphanet: ORPHA916

Definition

A very rare genetic disorder with characteristics of the following congenital malformations: hydrocephalus (due to Dandy-Walker anomaly), cleft palate and severe joint contractures. Less than 20 cases have been reported in the literature. The fingers are thin with absent knuckles and reduced creases over the joints and patients show an inability to make a full fist. Additional findings may include deformed ears, ptosis, an inability to open the mouth fully, heart defects, and clubfoot. There are currently no human genes associated with this disease. [from SNOMEDCT_US]

Clinical features

From HPO
Congenital neuroblastoma
MedGen UID:
870538
Concept ID:
C4024986
Neoplastic Process
Clubfoot
MedGen UID:
3130
Concept ID:
C0009081
Congenital Abnormality
Clubfoot is a congenital limb deformity defined as fixation of the foot in cavus, adductus, varus, and equinus (i.e., inclined inwards, axially rotated outwards, and pointing downwards) with concomitant soft tissue abnormalities (Cardy et al., 2007). Clubfoot may occur in isolation or as part of a syndrome (e.g., diastrophic dysplasia, 222600). Clubfoot has been reported with deficiency of long bones and mirror-image polydactyly (Gurnett et al., 2008; Klopocki et al., 2012).
Slender finger
MedGen UID:
387832
Concept ID:
C1857482
Finding
Fingers that are disproportionately narrow (reduced girth) for the hand/foot size or build of the individual.
Ventricular septal defect
MedGen UID:
42366
Concept ID:
C0018818
Congenital Abnormality
A hole between the two bottom chambers (ventricles) of the heart. The defect is centered around the most superior aspect of the ventricular septum.
Abnormal pinna morphology
MedGen UID:
167800
Concept ID:
C0857379
Congenital Abnormality
An abnormality of the pinna, which is also referred to as the auricle or external ear.
Hydrocephalus
MedGen UID:
9335
Concept ID:
C0020255
Disease or Syndrome
Hydrocephalus is an active distension of the ventricular system of the brain resulting from inadequate passage of CSF from its point of production within the cerebral ventricles to its point of absorption into the systemic circulation.
Dandy-Walker syndrome
MedGen UID:
4150
Concept ID:
C0010964
Disease or Syndrome
Dandy-Walker malformation is defined by hypoplasia and upward rotation of the cerebellar vermis and cystic dilation of the fourth ventricle. Affected individuals often have motor deficits such as delayed motor development, hypotonia, and ataxia; about half have mental retardation and some have hydrocephalus. DWM is a heterogeneous disorder. The low empiric recurrence risk of approximately 1 to 2% for nonsyndromic DWM suggests that mendelian inheritance is unlikely (summary by Murray et al., 1985).
Flexion contracture
MedGen UID:
83069
Concept ID:
C0333068
Anatomical Abnormality
A flexion contracture is a bent (flexed) joint that cannot be straightened actively or passively. It is thus a chronic loss of joint motion due to structural changes in muscle, tendons, ligaments, or skin that prevents normal movement of joints.
Open mouth
MedGen UID:
116104
Concept ID:
C0240379
Finding
A facial appearance characterized by a permanently or nearly permanently opened mouth.
Cleft palate
MedGen UID:
756015
Concept ID:
C2981150
Congenital Abnormality
Cleft palate is a developmental defect of the palate resulting from a failure of fusion of the palatine processes and manifesting as a separation of the roof of the mouth (soft and hard palate).
Ptosis
MedGen UID:
2287
Concept ID:
C0005745
Disease or Syndrome
The upper eyelid margin is positioned 3 mm or more lower than usual and covers the superior portion of the iris (objective); or, the upper lid margin obscures at least part of the pupil (subjective).

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVAase-Smith syndrome
Follow this link to review classifications for Aase-Smith syndrome in Orphanet.

Professional guidelines

PubMed

Wang S, Peng D
Int Heart J 2019 Jan 25;60(1):12-18. Epub 2018 Dec 5 doi: 10.1536/ihj.17-604. PMID: 30518714
Cherian L, Rosenberg JJ
Pediatr Rev 2010 Sep;31(9):385-7. doi: 10.1542/pir.31-9-385. PMID: 20810705

Recent clinical studies

Etiology

Wang S, Peng D
Int Heart J 2019 Jan 25;60(1):12-18. Epub 2018 Dec 5 doi: 10.1536/ihj.17-604. PMID: 30518714
D'Avanzo M, Santinelli R, Tolone C, D'Avanzo A, De Simone A, Pistoia V
Haematologica 2002 Nov;87(11):ECR34. PMID: 12414360

Diagnosis

Wang S, Peng D
Int Heart J 2019 Jan 25;60(1):12-18. Epub 2018 Dec 5 doi: 10.1536/ihj.17-604. PMID: 30518714
Cinotti E, Ferrero G, Paparo F, Papadia M, Faravelli F, Rongioletti F, Traverso C, Di Maria E
Am J Med Genet A 2013 Jun;161A(6):1214-20. Epub 2013 May 1 doi: 10.1002/ajmg.a.35908. PMID: 23637089
D'Avanzo M, Santinelli R, Tolone C, D'Avanzo A, De Simone A, Pistoia V
Haematologica 2002 Nov;87(11):ECR34. PMID: 12414360
David A, Nombalais MF, Rival JM, Verloes A
Clin Genet 1996 Oct;50(4):251-4. doi: 10.1111/j.1399-0004.1996.tb02638.x. PMID: 9001811
Yetgin S, Balci S, Irken G, Coşkun T, Say B
Turk J Pediatr 1994 Jul-Sep;36(3):239-42. PMID: 7974814

Therapy

Wang S, Peng D
Int Heart J 2019 Jan 25;60(1):12-18. Epub 2018 Dec 5 doi: 10.1536/ihj.17-604. PMID: 30518714
Soker M, Ayyildiz O, Isikdogan A
Saudi Med J 2004 Dec;25(12):2004-6. PMID: 15711686

Prognosis

D'Avanzo M, Santinelli R, Tolone C, D'Avanzo A, De Simone A, Pistoia V
Haematologica 2002 Nov;87(11):ECR34. PMID: 12414360

Clinical prediction guides

D'Avanzo M, Pistoia V, Santinelli R, Tolone C, Toraldo R, Corcione A, Canino G, Iafusco F
Pediatr Hematol Oncol 1994 Mar-Apr;11(2):189-95. doi: 10.3109/08880019409141655. PMID: 8204444

Supplemental Content

Table of contents

    Clinical resources

    Practice guidelines

    • PubMed
      See practice and clinical guidelines in PubMed. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.

    Recent activity

    Your browsing activity is empty.

    Activity recording is turned off.

    Turn recording back on

    See more...