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Emery-Dreifuss muscular dystrophy 4, autosomal dominant(EDMD4)

MedGen UID:
414476
Concept ID:
C2751807
Disease or Syndrome
Synonyms: EDMD4; EMERY-DREIFUSS MUSCULAR DYSTROPHY 4 WITH VARIABLE FEATURES; SYNE1-Related Emery-Dreifuss Muscular Dystrophy
 
Gene (location): SYNE1 (6q25.2)
 
Monarch Initiative: MONDO:0013071
OMIM®: 612998

Definition

Any autosomal dominant Emery-Dreifuss muscular dystrophy in which the cause of the disease is a mutation in the SYNE1 gene. [from MONDO]

Clinical features

From HPO
Ventricular septal hypertrophy
MedGen UID:
138013
Concept ID:
C0344955
Finding
The dividing wall between left and right sides of the heart, thickens and bulges into the left ventricle.
Muscular dystrophy
MedGen UID:
44527
Concept ID:
C0026850
Disease or Syndrome
The term dystrophy means abnormal growth. However, muscular dystrophy is used to describe primary myopathies with a genetic basis and a progressive course characterized by progressive skeletal muscle weakness and wasting, defects in muscle proteins, and histological features of muscle fiber degeneration (necrosis) and regeneration. If possible, it is preferred to use other HPO terms to describe the precise phenotypic abnormalities.
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.
Proximal amyotrophy
MedGen UID:
342591
Concept ID:
C1850794
Disease or Syndrome
Amyotrophy (muscular atrophy) affecting the proximal musculature.
Elevated circulating creatine kinase concentration
MedGen UID:
69128
Concept ID:
C0241005
Finding
An elevation of the level of the enzyme creatine kinase (also known as creatine phosphokinase (CK; EC 2.7.3.2) in the blood. CK levels can be elevated in a number of clinical disorders such as myocardial infarction, rhabdomyolysis, and muscular dystrophy.

Recent clinical studies

Etiology

Maggi L, D'Amico A, Pini A, Sivo S, Pane M, Ricci G, Vercelli L, D'Ambrosio P, Travaglini L, Sala S, Brenna G, Kapetis D, Scarlato M, Pegoraro E, Ferrari M, Toscano A, Benedetti S, Bernasconi P, Colleoni L, Lattanzi G, Bertini E, Mercuri E, Siciliano G, Rodolico C, Mongini T, Politano L, Previtali SC, Carboni N, Mantegazza R, Morandi L
Neurology 2014 Oct 28;83(18):1634-44. Epub 2014 Oct 1 doi: 10.1212/WNL.0000000000000934. PMID: 25274841
Mercuri E, Clements E, Offiah A, Pichiecchio A, Vasco G, Bianco F, Berardinelli A, Manzur A, Pane M, Messina S, Gualandi F, Ricci E, Rutherford M, Muntoni F
Ann Neurol 2010 Feb;67(2):201-8. doi: 10.1002/ana.21846. PMID: 20225280
Smith GC, Kinali M, Prasad SK, Bonne G, Muntoni F, Pennell DJ, Nihoyannopoulos P
J Cardiovasc Magn Reson 2006;8(5):723-30. doi: 10.1080/10976640600723862. PMID: 16891232
Sinagra G, Di Lenarda A, Brodsky GL, Taylor MR, Muntoni F, Pinamonti B, Carniel E, Driussi M, Bristow MR, Mestroni L; Heart Muscle Disease Study Group
Ital Heart J 2001 Apr;2(4):280-6. PMID: 11374497
Yates JR, Warner JP, Smith JA, Deymeer F, Azulay JP, Hausmanowa-Petrusewicz I, Zaremba J, Borkowska J, Affara NA, Ferguson-Smith MA
J Med Genet 1993 Feb;30(2):108-11. doi: 10.1136/jmg.30.2.108. PMID: 8445613Free PMC Article

Diagnosis

Le Thanh P, Meinke P, Korfali N, Srsen V, Robson MI, Wehnert M, Schoser B, Sewry CA, Schirmer EC
Neuromuscul Disord 2017 Apr;27(4):338-351. Epub 2016 Dec 21 doi: 10.1016/j.nmd.2016.12.003. PMID: 28214269Free PMC Article
Orrell RW
Handb Clin Neurol 2011;101:167-80. doi: 10.1016/B978-0-08-045031-5.00013-X. PMID: 21496633
Niebroj-Dobosz I, Madej-Pilarczyk A, Marchel M, Sokołowska B, Hausmanowa-Petrusewicz I
Acta Biochim Pol 2009;56(4):717-22. Epub 2009 Dec 8 PMID: 19997654
Sewry CA, Brown SC, Mercuri E, Bonne G, Feng L, Camici G, Morris GE, Muntoni F
Neuropathol Appl Neurobiol 2001 Aug;27(4):281-90. doi: 10.1046/j.0305-1846.2001.00323.x. PMID: 11532159
Sinagra G, Di Lenarda A, Brodsky GL, Taylor MR, Muntoni F, Pinamonti B, Carniel E, Driussi M, Bristow MR, Mestroni L; Heart Muscle Disease Study Group
Ital Heart J 2001 Apr;2(4):280-6. PMID: 11374497

Therapy

Smith GC, Kinali M, Prasad SK, Bonne G, Muntoni F, Pennell DJ, Nihoyannopoulos P
J Cardiovasc Magn Reson 2006;8(5):723-30. doi: 10.1080/10976640600723862. PMID: 16891232
Boriani G, Gallina M, Merlini L, Bonne G, Toniolo D, Amati S, Biffi M, Martignani C, Frabetti L, Bonvicini M, Rapezzi C, Branzi A
Stroke 2003 Apr;34(4):901-8. Epub 2003 Mar 20 doi: 10.1161/01.STR.0000064322.47667.49. PMID: 12649505

Prognosis

Orrell RW
Handb Clin Neurol 2011;101:167-80. doi: 10.1016/B978-0-08-045031-5.00013-X. PMID: 21496633
Niebroj-Dobosz I, Madej-Pilarczyk A, Marchel M, Sokołowska B, Hausmanowa-Petrusewicz I
Acta Biochim Pol 2009;56(4):717-22. Epub 2009 Dec 8 PMID: 19997654
Smith GC, Kinali M, Prasad SK, Bonne G, Muntoni F, Pennell DJ, Nihoyannopoulos P
J Cardiovasc Magn Reson 2006;8(5):723-30. doi: 10.1080/10976640600723862. PMID: 16891232
Boriani G, Gallina M, Merlini L, Bonne G, Toniolo D, Amati S, Biffi M, Martignani C, Frabetti L, Bonvicini M, Rapezzi C, Branzi A
Stroke 2003 Apr;34(4):901-8. Epub 2003 Mar 20 doi: 10.1161/01.STR.0000064322.47667.49. PMID: 12649505
Sinagra G, Di Lenarda A, Brodsky GL, Taylor MR, Muntoni F, Pinamonti B, Carniel E, Driussi M, Bristow MR, Mestroni L; Heart Muscle Disease Study Group
Ital Heart J 2001 Apr;2(4):280-6. PMID: 11374497

Clinical prediction guides

Le Thanh P, Meinke P, Korfali N, Srsen V, Robson MI, Wehnert M, Schoser B, Sewry CA, Schirmer EC
Neuromuscul Disord 2017 Apr;27(4):338-351. Epub 2016 Dec 21 doi: 10.1016/j.nmd.2016.12.003. PMID: 28214269Free PMC Article
Burke B, Mounkes LC, Stewart CL
Traffic 2001 Oct;2(10):675-83. doi: 10.1034/j.1600-0854.2001.21001.x. PMID: 11576443
Sewry CA, Brown SC, Mercuri E, Bonne G, Feng L, Camici G, Morris GE, Muntoni F
Neuropathol Appl Neurobiol 2001 Aug;27(4):281-90. doi: 10.1046/j.0305-1846.2001.00323.x. PMID: 11532159
Sinagra G, Di Lenarda A, Brodsky GL, Taylor MR, Muntoni F, Pinamonti B, Carniel E, Driussi M, Bristow MR, Mestroni L; Heart Muscle Disease Study Group
Ital Heart J 2001 Apr;2(4):280-6. PMID: 11374497
Yates JR, Warner JP, Smith JA, Deymeer F, Azulay JP, Hausmanowa-Petrusewicz I, Zaremba J, Borkowska J, Affara NA, Ferguson-Smith MA
J Med Genet 1993 Feb;30(2):108-11. doi: 10.1136/jmg.30.2.108. PMID: 8445613Free PMC Article

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