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Congenital muscular dystrophy merosin-positive

MedGen UID:
322832
Concept ID:
C1836133
Disease or Syndrome
Synonym: Muscular Dystrophy, Congenital, Merosin-Positive
 
OMIM®: 609456

Clinical features

From HPO
Shoulder girdle muscle weakness
MedGen UID:
96533
Concept ID:
C0427063
Finding
The shoulder, or pectoral, girdle is composed of the clavicles and the scapulae. Shoulder-girdle weakness refers to lack of strength of the muscles attaching to these bones, that is, lack of strength of the muscles around the shoulders.
Delayed ability to walk
MedGen UID:
66034
Concept ID:
C0241726
Finding
A failure to achieve the ability to walk at an appropriate developmental stage. Most children learn to walk in a series of stages, and learn to walk short distances independently between 12 and 15 months.
Myopathy
MedGen UID:
10135
Concept ID:
C0026848
Disease or Syndrome
A disorder of muscle unrelated to impairment of innervation or neuromuscular junction.
Scoliosis
MedGen UID:
11348
Concept ID:
C0036439
Disease or Syndrome
The presence of an abnormal lateral curvature of the spine.
Joint laxity
MedGen UID:
39439
Concept ID:
C0086437
Finding
Lack of stability of a joint.
Proximal muscle weakness
MedGen UID:
113169
Concept ID:
C0221629
Finding
A lack of strength of the proximal muscles.
Neck muscle weakness
MedGen UID:
66808
Concept ID:
C0240479
Finding
Decreased strength of the neck musculature.
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.
Facial palsy
MedGen UID:
87660
Concept ID:
C0376175
Disease or Syndrome
Facial nerve palsy is a dysfunction of cranial nerve VII (the facial nerve) that results in inability to control facial muscles on the affected side with weakness of the muscles of facial expression and eye closure. This can either be present in unilateral or bilateral form.
Congenital muscular dystrophy
MedGen UID:
147063
Concept ID:
C0699743
Disease or Syndrome
Congenital muscular dystrophy (CMD) is a heterogeneous group of neuromuscular disorders with onset at birth or infancy characterized by hypotonia, muscle wasting, weakness or delayed motor milestones. The group includes myopathies with abnormalities at different cellular levels: the extracellular matrix (MDC1A, UCMD; see these terms), the dystrophin-associated glycoprotein complex (alphadystroglycanopathies, integrinopathies see these terms), the endoplasmic reticulum (rigid spine syndrome [RSMD1], and the nuclear envelope (LMNA-related CMD; [L-CMD] and Nesprin-1-related CMD; see these terms).
Increased variability in muscle fiber diameter
MedGen UID:
336019
Concept ID:
C1843700
Finding
An abnormally high degree of muscle fiber size variation. This phenotypic feature can be observed upon muscle biopsy.
Neonatal hypotonia
MedGen UID:
412209
Concept ID:
C2267233
Disease or Syndrome
Muscular hypotonia (abnormally low muscle tone) manifesting in the neonatal period.
Respiratory insufficiency due to muscle weakness
MedGen UID:
812797
Concept ID:
C3806467
Finding
Mildly elevated creatine kinase
MedGen UID:
342469
Concept ID:
C1850309
Finding
Decreased fetal movement
MedGen UID:
68618
Concept ID:
C0235659
Finding
An abnormal reduction in quantity or strength of fetal movements.

Professional guidelines

PubMed

Klein A, Clement E, Mercuri E, Muntoni F
Eur J Paediatr Neurol 2008 Sep;12(5):371-7. Epub 2007 Dec 3 doi: 10.1016/j.ejpn.2007.10.002. PMID: 18588847

Recent clinical studies

Etiology

Quijano-Roy S, Renault F, Romero N, Guicheney P, Fardeau M, Estournet B
Muscle Nerve 2004 Feb;29(2):292-9. doi: 10.1002/mus.10544. PMID: 14755496
Flanigan KM, Kerr L, Bromberg MB, Leonard C, Tsuruda J, Zhang P, Gonzalez-Gomez I, Cohn R, Campbell KP, Leppert M
Ann Neurol 2000 Feb;47(2):152-61. PMID: 10665485
Mercuri E, Anker S, Philpot J, Sewry C, Dubowitz V, Muntoni F
Pediatr Neurol 1998 May;18(5):399-401. doi: 10.1016/s0887-8994(97)00222-1. PMID: 9650678
Nonaka I, Kobayashi O, Osari S
Semin Pediatr Neurol 1996 Jun;3(2):110-21. doi: 10.1016/s1071-9091(96)80040-4. PMID: 8795845
Shorer Z, Philpot J, Muntoni F, Sewry C, Dubowitz V
J Child Neurol 1995 Nov;10(6):472-5. doi: 10.1177/088307389501000610. PMID: 8576559

Diagnosis

Fadiloglu E, Ozten G, Unal C, Talim B, Topaloglu H, Beksac MS
Fetal Pediatr Pathol 2018 Dec;37(6):418-423. Epub 2018 Oct 25 doi: 10.1080/15513815.2018.1520944. PMID: 30358464
Talim B, Kale G, Topaloglu H, Akçören Z, Caglar M, Gögüş S, Elkay M
Pediatr Dev Pathol 2000 Mar-Apr;3(2):168-76. doi: 10.1007/s100240050021. PMID: 10679036
Mahjneh I, Bushby K, Anderson L, Muntoni F, Tolvanen-Mahjneh H, Bashir R, Pizzi A, Brockington M, Marconi G
Neuropediatrics 1999 Feb;30(1):22-8. doi: 10.1055/s-2007-973452. PMID: 10222457
Mercuri E, Anker S, Philpot J, Sewry C, Dubowitz V, Muntoni F
Pediatr Neurol 1998 May;18(5):399-401. doi: 10.1016/s0887-8994(97)00222-1. PMID: 9650678
Nonaka I, Kobayashi O, Osari S
Semin Pediatr Neurol 1996 Jun;3(2):110-21. doi: 10.1016/s1071-9091(96)80040-4. PMID: 8795845

Prognosis

Ferreira LG, Marie SK, Liu EC, Resende MB, Carvalho MS, Scaff M, Reed UC
Arq Neuropsiquiatr 2005 Sep;63(3B):791-800. Epub 2005 Oct 18 doi: 10.1590/s0004-282x2005000500014. PMID: 16258658
Ceviz N, Alehan F, Alehan D, Ozme S, Akçören Z, Kale G, Topaloglu H
Int J Cardiol 2003 Feb;87(2-3):129-33; discussion 133-4. doi: 10.1016/s0167-5273(02)00320-0. PMID: 12559529
Flanigan KM, Kerr L, Bromberg MB, Leonard C, Tsuruda J, Zhang P, Gonzalez-Gomez I, Cohn R, Campbell KP, Leppert M
Ann Neurol 2000 Feb;47(2):152-61. PMID: 10665485
Mahjneh I, Bushby K, Anderson L, Muntoni F, Tolvanen-Mahjneh H, Bashir R, Pizzi A, Brockington M, Marconi G
Neuropediatrics 1999 Feb;30(1):22-8. doi: 10.1055/s-2007-973452. PMID: 10222457
Connolly AM, Pestronk A, Planer GJ, Yue J, Mehta S, Choksi R
Neurology 1996 Mar;46(3):810-14. doi: 10.1212/wnl.46.3.810. PMID: 8618688

Clinical prediction guides

Quijano-Roy S, Renault F, Romero N, Guicheney P, Fardeau M, Estournet B
Muscle Nerve 2004 Feb;29(2):292-9. doi: 10.1002/mus.10544. PMID: 14755496
Flanigan KM, Kerr L, Bromberg MB, Leonard C, Tsuruda J, Zhang P, Gonzalez-Gomez I, Cohn R, Campbell KP, Leppert M
Ann Neurol 2000 Feb;47(2):152-61. PMID: 10665485
Mercuri E, Gruter-Andrew J, Philpot J, Sewry C, Counsell S, Henderson S, Jensen A, Naom I, Bydder G, Dubowitz V, Muntoni F
Neuromuscul Disord 1999 Oct;9(6-7):383-7. doi: 10.1016/s0960-8966(99)00034-6. PMID: 10545041
Mercuri E, Anker S, Philpot J, Sewry C, Dubowitz V, Muntoni F
Pediatr Neurol 1998 May;18(5):399-401. doi: 10.1016/s0887-8994(97)00222-1. PMID: 9650678
Connolly AM, Pestronk A, Planer GJ, Yue J, Mehta S, Choksi R
Neurology 1996 Mar;46(3):810-14. doi: 10.1212/wnl.46.3.810. PMID: 8618688

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