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Myopathy, congenital, with tremor(MYOTREM; CMYP16)

MedGen UID:
1684886
Concept ID:
C5231401
Disease or Syndrome
Synonyms: CONGENITAL MYOPATHY 16; MYOGENIC TREMOR
 
Gene (location): MYBPC1 (12q23.2)
 
Monarch Initiative: MONDO:0032797
OMIM®: 618524

Definition

Congenital myopathy-16 (CMYO16) is an autosomal dominant muscle disorder characterized by onset of hypotonia and tremor in infancy. Patients have mildly delayed walking, unsteady gait, proximal muscle weakness, and a high-frequency tremor of the limbs. Some may develop secondary mild contractures or spinal deformities. Cognition is normal and the disease course tends to stabilize after adolescence (summary by Stavusis et al., 2019). For a discussion of genetic heterogeneity of congenital myopathy, see CMYO1A (117000). [from OMIM]

Clinical features

From HPO
Scapular winging
MedGen UID:
66822
Concept ID:
C0240953
Anatomical Abnormality
Abnormal protrusion of the scapula away from the surface of the back.
Postural tremor
MedGen UID:
66696
Concept ID:
C0234378
Sign or Symptom
A type of tremors that is triggered by holding a limb in a fixed position.
Tongue tremor
MedGen UID:
1630956
Concept ID:
C0241446
Finding
An unintentional, oscillating to-and-fro muscle movement affecting the tongue.
Micrognathia
MedGen UID:
44428
Concept ID:
C0025990
Congenital Abnormality
Developmental hypoplasia of the mandible.
Scoliosis
MedGen UID:
11348
Concept ID:
C0036439
Disease or Syndrome
The presence of an abnormal lateral curvature of the spine.
Proximal muscle weakness
MedGen UID:
113169
Concept ID:
C0221629
Finding
A lack of strength of the proximal muscles.
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.
Distal muscle weakness
MedGen UID:
140883
Concept ID:
C0427065
Finding
Reduced strength of the musculature of the distal extremities.
Lumbar hyperlordosis
MedGen UID:
263149
Concept ID:
C1184923
Finding
An abnormal accentuation of the inward curvature of the spine in the lumbar region.
Axial muscle weakness
MedGen UID:
334472
Concept ID:
C1843697
Finding
Reduced strength of the axial musculature (i.e., of the muscles of the head and neck, spine, and ribs).
Spinal rigidity
MedGen UID:
346721
Concept ID:
C1858025
Finding
Reduced ability to move the vertebral column with a resulting limitation of neck and trunk flexion.
EMG: myopathic abnormalities
MedGen UID:
867362
Concept ID:
C4021726
Pathologic Function
The presence of abnormal electromyographic patterns indicative of myopathy, such as small-short polyphasic motor unit potentials.
Narrow mouth
MedGen UID:
44435
Concept ID:
C0026034
Congenital Abnormality
Distance between the commissures of the mouth more than 2 SD below the mean. Alternatively, an apparently decreased width of the oral aperture (subjective).
High palate
MedGen UID:
66814
Concept ID:
C0240635
Congenital Abnormality
Height of the palate more than 2 SD above the mean (objective) or palatal height at the level of the first permanent molar more than twice the height of the teeth (subjective).
Prominent nasolabial fold
MedGen UID:
355725
Concept ID:
C1866487
Finding
Exaggerated bulkiness of the crease or fold of skin running from the lateral margin of the nose, where nasal base meets the skin of the face, to a point just lateral to the corner of the mouth (cheilion, or commissure).

Recent clinical studies

Etiology

Stavusis J, Geist J, Kontrogianni-Konstantopoulos A
J Muscle Res Cell Motil 2020 Dec;41(4):285-295. Epub 2019 Oct 16 doi: 10.1007/s10974-019-09559-1. PMID: 31620961Free PMC Article

Diagnosis

Stavusis J, Geist J, Kontrogianni-Konstantopoulos A
J Muscle Res Cell Motil 2020 Dec;41(4):285-295. Epub 2019 Oct 16 doi: 10.1007/s10974-019-09559-1. PMID: 31620961Free PMC Article
Marra JD, Engelstad KE, Ankala A, Tanji K, Dastgir J, De Vivo DC, Coffee B, Chiriboga CA
Muscle Nerve 2015 May;51(5):767-72. Epub 2015 Feb 17 doi: 10.1002/mus.24528. PMID: 25430424
Vaamonde J, Artieda J, Obeso JA
Mov Disord 1991;6(2):180-2. doi: 10.1002/mds.870060218. PMID: 1647493

Therapy

Streib EW
Ann Neurol 1986 May;19(5):501-4. doi: 10.1002/ana.410190515. PMID: 3087270

Prognosis

Iyer A, Lauerova B, Mariano J, Haberlová J, Lassuthova P, Zidkova J, Wright NT, Kontrogianni-Konstantopoulos A
Gene 2024 Jun 5;910:148339. Epub 2024 Mar 2 doi: 10.1016/j.gene.2024.148339. PMID: 38438057Free PMC Article

Clinical prediction guides

Iyer A, Lauerova B, Mariano J, Haberlová J, Lassuthova P, Zidkova J, Wright NT, Kontrogianni-Konstantopoulos A
Gene 2024 Jun 5;910:148339. Epub 2024 Mar 2 doi: 10.1016/j.gene.2024.148339. PMID: 38438057Free PMC Article
Dommergues M, Candilis D, Becerra L, Thoueille E, Cohen D, Viaux-Savelon S
Orphanet J Rare Dis 2021 Apr 13;16(1):176. doi: 10.1186/s13023-021-01810-8. PMID: 33849607Free PMC Article
Stavusis J, Geist J, Kontrogianni-Konstantopoulos A
J Muscle Res Cell Motil 2020 Dec;41(4):285-295. Epub 2019 Oct 16 doi: 10.1007/s10974-019-09559-1. PMID: 31620961Free PMC Article
Vaamonde J, Artieda J, Obeso JA
Mov Disord 1991;6(2):180-2. doi: 10.1002/mds.870060218. PMID: 1647493

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