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Congenital muscular dystrophy due to partial LAMA2 deficiency

MedGen UID:
335826
Concept ID:
C1842898
Disease or Syndrome
Synonym: Muscular Dystrophy, Congenital, due to Partial LAMA2 Deficiency
 
OMIM®: 156225; 607855

Definition

Symptoms of late-onset LAMA2-related muscular dystrophy become evident later in childhood or adulthood, and are similar to those of a group of muscle disorders classified as limb-girdle muscular dystrophies. In late-onset LAMA2-related muscular dystrophy, the muscles most affected are those closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area, and thighs. Children with late-onset LAMA2-related muscular dystrophy sometimes have delayed development of motor skills such as walking, but generally achieve the ability to walk without assistance. Over time, they may develop rigidity of the back, joint contractures, scoliosis, and breathing problems. However, most affected individuals retain the ability to walk and climb stairs.

As affected children grow, they often develop an abnormal, gradually worsening side-to-side curvature of the spine (scoliosis) and inward curvature of the back (lordosis). Children with early-onset LAMA2-related muscular dystrophy often do not develop the ability to walk. Difficulty with speech may result from weakness of the facial muscles and an enlarged tongue. Seizures occur in about a third of individuals with early-onset LAMA2-related muscular dystrophy; rarely, heart complications occur in this form of the disorder.

Early-onset LAMA2-related muscular dystrophy is apparent at birth or within the first few months of life. It is considered part of a class of muscle disorders called congenital muscular dystrophies and is sometimes called congenital muscular dystrophy type 1A. Affected infants may have severe muscle weakness, lack of muscle tone (hypotonia), little spontaneous movement, and joint deformities (contractures). Weakness of the muscles in the face and throat can result in feeding difficulties and an inability to grow and gain weight at the expected rate. Respiratory insufficiency, which occurs when muscles in the chest are weakened, causes a weak cry and breathing problems that can lead to frequent, potentially life-threatening lung infections.

LAMA2-related muscular dystrophy is a disorder that causes weakness and wasting (atrophy) of muscles used for movement (skeletal muscles). This condition varies in severity, from a severe, early-onset type to a milder, late-onset form. [from MedlinePlus Genetics]

Term Hierarchy

Professional guidelines

PubMed

Xiong H, Tan D, Wang S, Song S, Yang H, Gao K, Liu A, Jiao H, Mao B, Ding J, Chang X, Wang J, Wu Y, Yuan Y, Jiang Y, Zhang F, Wu H, Wu X
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Arch Neurol 2005 Oct;62(10):1582-6. doi: 10.1001/archneur.62.10.1582. PMID: 16216942
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Recent clinical studies

Etiology

Quijano-Roy S, Haberlova J, Castiglioni C, Vissing J, Munell F, Rivier F, Stojkovic T, Malfatti E, Gómez García de la Banda M, Tasca G, Costa Comellas L, Benezit A, Amthor H, Dabaj I, Gontijo Camelo C, Laforêt P, Rendu J, Romero NB, Cavassa E, Fattori F, Beroud C, Zídková J, Leboucq N, Løkken N, Sanchez-Montañez Á, Ortega X, Kynčl M, Metay C, Gómez-Andrés D, Carlier RY
J Neurol 2022 May;269(5):2414-2429. Epub 2021 Sep 24 doi: 10.1007/s00415-021-10806-0. PMID: 34559299
Bouman K, Groothuis JT, Doorduin J, van Alfen N, Udink Ten Cate FEA, van den Heuvel FMA, Nijveldt R, van Tilburg WCM, Buckens SCFM, Dittrich ATM, Draaisma JMT, Janssen MCH, Kamsteeg EJ, van Kleef ESB, Koene S, Smeitink JAM, Küsters B, van Tienen FHJ, Smeets HJM, van Engelen BGM, Erasmus CE, Voermans NC
BMC Neurol 2021 Aug 12;21(1):313. doi: 10.1186/s12883-021-02336-z. PMID: 34384384Free PMC Article
Camacho A, Núñez N, Dekomien G, Hernández-Laín A, de Aragón AM, Simón R
Eur J Paediatr Neurol 2015 Mar;19(2):243-7. Epub 2014 Dec 2 doi: 10.1016/j.ejpn.2014.11.005. PMID: 25500573
Triki C, Louhichi N, Méziou M, Choyakh F, Kéchaou MS, Jlidi R, Mhiri C, Fakhfakh F, Ayadi H
Neuromuscul Disord 2003 Jan;13(1):4-12. doi: 10.1016/s0960-8966(02)00188-8. PMID: 12467726
Naom I, D'Alessandro M, Sewry C, Ferlini A, Topaloglu H, Helbling-Leclerc A, Guicheney P, Schwartz K, Akcoren Z, Dubowitz V, Muntoni F
Hum Genet 1997 Apr;99(4):535-40. doi: 10.1007/s004390050402. PMID: 9099847

Diagnosis

Bouman K, Groothuis JT, Doorduin J, van Alfen N, Udink Ten Cate FEA, van den Heuvel FMA, Nijveldt R, van Tilburg WCM, Buckens SCFM, Dittrich ATM, Draaisma JMT, Janssen MCH, Kamsteeg EJ, van Kleef ESB, Koene S, Smeitink JAM, Küsters B, van Tienen FHJ, Smeets HJM, van Engelen BGM, Erasmus CE, Voermans NC
BMC Neurol 2021 Aug 12;21(1):313. doi: 10.1186/s12883-021-02336-z. PMID: 34384384Free PMC Article
Specht S, Duff J, Charlton R, Polvikoski T, Barresi R, Töpf A, Straub V
Neuromuscul Disord 2021 Jul;31(7):660-665. Epub 2021 Apr 1 doi: 10.1016/j.nmd.2021.03.009. PMID: 34074572
Natera-de Benito D, Muchart J, Itzep D, Ortez C, González-Quereda L, Gallano P, Ramirez A, Aparicio J, Domínguez-Carral J, Carrera-García L, Expósito-Escudero J, Pardo Cardozo N, Cuadras D, Codina A, Jou C, Jimenez-Mallebrera C, Palau F, Colomer J, Arzimanoglou A, Nascimento A, San Antonio-Arce V
Epilepsia 2020 May;61(5):971-983. Epub 2020 Apr 8 doi: 10.1111/epi.16493. PMID: 32266982
Xiong H, Tan D, Wang S, Song S, Yang H, Gao K, Liu A, Jiao H, Mao B, Ding J, Chang X, Wang J, Wu Y, Yuan Y, Jiang Y, Zhang F, Wu H, Wu X
Clin Genet 2015 Mar;87(3):233-43. Epub 2014 Mar 31 doi: 10.1111/cge.12366. PMID: 24611677
Di Blasi C, Piga D, Brioschi P, Moroni I, Pini A, Ruggieri A, Zanotti S, Uziel G, Jarre L, Della Giustina E, Scuderi C, Jonsrud C, Mantegazza R, Morandi L, Mora M
Arch Neurol 2005 Oct;62(10):1582-6. doi: 10.1001/archneur.62.10.1582. PMID: 16216942

Therapy

Bouman K, Groothuis JT, Doorduin J, van Alfen N, Udink Ten Cate FEA, van den Heuvel FMA, Nijveldt R, van Tilburg WCM, Buckens SCFM, Dittrich ATM, Draaisma JMT, Janssen MCH, Kamsteeg EJ, van Kleef ESB, Koene S, Smeitink JAM, Küsters B, van Tienen FHJ, Smeets HJM, van Engelen BGM, Erasmus CE, Voermans NC
BMC Neurol 2021 Aug 12;21(1):313. doi: 10.1186/s12883-021-02336-z. PMID: 34384384Free PMC Article
Natera-de Benito D, Muchart J, Itzep D, Ortez C, González-Quereda L, Gallano P, Ramirez A, Aparicio J, Domínguez-Carral J, Carrera-García L, Expósito-Escudero J, Pardo Cardozo N, Cuadras D, Codina A, Jou C, Jimenez-Mallebrera C, Palau F, Colomer J, Arzimanoglou A, Nascimento A, San Antonio-Arce V
Epilepsia 2020 May;61(5):971-983. Epub 2020 Apr 8 doi: 10.1111/epi.16493. PMID: 32266982
Camacho A, Núñez N, Dekomien G, Hernández-Laín A, de Aragón AM, Simón R
Eur J Paediatr Neurol 2015 Mar;19(2):243-7. Epub 2014 Dec 2 doi: 10.1016/j.ejpn.2014.11.005. PMID: 25500573
Yamauchi J, Kumar A, Duarte L, Mehuron T, Girgenrath M
Hum Mol Genet 2013 Nov 1;22(21):4306-17. Epub 2013 Jun 16 doi: 10.1093/hmg/ddt280. PMID: 23773998

Prognosis

Specht S, Duff J, Charlton R, Polvikoski T, Barresi R, Töpf A, Straub V
Neuromuscul Disord 2021 Jul;31(7):660-665. Epub 2021 Apr 1 doi: 10.1016/j.nmd.2021.03.009. PMID: 34074572
Natera-de Benito D, Muchart J, Itzep D, Ortez C, González-Quereda L, Gallano P, Ramirez A, Aparicio J, Domínguez-Carral J, Carrera-García L, Expósito-Escudero J, Pardo Cardozo N, Cuadras D, Codina A, Jou C, Jimenez-Mallebrera C, Palau F, Colomer J, Arzimanoglou A, Nascimento A, San Antonio-Arce V
Epilepsia 2020 May;61(5):971-983. Epub 2020 Apr 8 doi: 10.1111/epi.16493. PMID: 32266982
Cohn RD, Herrmann R, Sorokin L, Wewer UM, Voit T
Neurology 1998 Jul;51(1):94-100. doi: 10.1212/wnl.51.1.94. PMID: 9674785

Clinical prediction guides

Quijano-Roy S, Haberlova J, Castiglioni C, Vissing J, Munell F, Rivier F, Stojkovic T, Malfatti E, Gómez García de la Banda M, Tasca G, Costa Comellas L, Benezit A, Amthor H, Dabaj I, Gontijo Camelo C, Laforêt P, Rendu J, Romero NB, Cavassa E, Fattori F, Beroud C, Zídková J, Leboucq N, Løkken N, Sanchez-Montañez Á, Ortega X, Kynčl M, Metay C, Gómez-Andrés D, Carlier RY
J Neurol 2022 May;269(5):2414-2429. Epub 2021 Sep 24 doi: 10.1007/s00415-021-10806-0. PMID: 34559299
Specht S, Duff J, Charlton R, Polvikoski T, Barresi R, Töpf A, Straub V
Neuromuscul Disord 2021 Jul;31(7):660-665. Epub 2021 Apr 1 doi: 10.1016/j.nmd.2021.03.009. PMID: 34074572
Natera-de Benito D, Muchart J, Itzep D, Ortez C, González-Quereda L, Gallano P, Ramirez A, Aparicio J, Domínguez-Carral J, Carrera-García L, Expósito-Escudero J, Pardo Cardozo N, Cuadras D, Codina A, Jou C, Jimenez-Mallebrera C, Palau F, Colomer J, Arzimanoglou A, Nascimento A, San Antonio-Arce V
Epilepsia 2020 May;61(5):971-983. Epub 2020 Apr 8 doi: 10.1111/epi.16493. PMID: 32266982
Rajakulendran S, Parton M, Holton JL, Hanna MG
Muscle Nerve 2011 Oct;44(4):590-3. doi: 10.1002/mus.22196. PMID: 21922472
Cohn RD, Herrmann R, Sorokin L, Wewer UM, Voit T
Neurology 1998 Jul;51(1):94-100. doi: 10.1212/wnl.51.1.94. PMID: 9674785

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