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Oculopharyngodistal myopathy(OPDM; FOLP-DR)

MedGen UID:
320250
Concept ID:
C1834014
Disease or Syndrome
Synonym: FACIOOCULOLARYNGOPHARYNGEAL MYOPATHY WITH DISTAL AND RESPIRATORY INVOLVEMENT
SNOMED CT: Oculopharyngeal distal myopathy (763829004); Oculopharyngodistal myopathy (763829004); OPDM - oculopharyngodistal myopathy (763829004)
Modes of inheritance:
Autosomal recessive inheritance
MedGen UID:
141025
Concept ID:
C0441748
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 individuals with two pathogenic alleles, either homozygotes (two copies of the same mutant allele) or compound heterozygotes (whereby each copy of a gene has a distinct mutant allele).
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.
 
Related genes: NOTCH2NLC, LRP12, GIPC1
 
Monarch Initiative: MONDO:0025193
OMIM®: 164310
OMIM® Phenotypic series: PS164310
Orphanet: ORPHA98897

Definition

A rare genetic neuromuscular disease with characteristics of progressive external ocular, facial and pharyngeal muscle weakness, leading to variable degrees of ptosis, ophthalmoparesis, facial muscle atrophy, dysarthria and dysphagia, as well as distal muscle weakness and atrophy of lower and upper extremities. Respiratory muscle involvement is common, but sensorineural hearing loss, asymmetrical extremity weakness and severe proximal weakness are rare. [from SNOMEDCT_US]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
Follow this link to review classifications for Oculopharyngodistal myopathy in Orphanet.

Professional guidelines

PubMed

Fan Y, Xu Y, Shi C
J Med Genet 2022 Jan;59(1):1-9. Epub 2021 Oct 21 doi: 10.1136/jmedgenet-2021-107883. PMID: 34675123
Nicolau S, Milone M, Liewluck T
Muscle Nerve 2021 Sep;64(3):255-269. Epub 2021 Jun 16 doi: 10.1002/mus.27337. PMID: 34133031

Recent clinical studies

Etiology

Eura N, Noguchi S, Ogasawara M, Kumutpongpanich T, Hayashi S, Nishino I; OPDM/OPMD Image Study Group
J Neurol 2023 Dec;270(12):5988-5998. Epub 2023 Aug 27 doi: 10.1007/s00415-023-11906-9. PMID: 37634163
Zhou ZD, Jankovic J, Ashizawa T, Tan EK
Nat Rev Neurol 2022 Mar;18(3):145-157. Epub 2022 Jan 12 doi: 10.1038/s41582-021-00612-7. PMID: 35022573
Cao L, Yan Y, Zhao G
Neurol Sci 2021 Oct;42(10):4055-4062. Epub 2021 Aug 1 doi: 10.1007/s10072-021-05498-3. PMID: 34333668
Kumutpongpanich T, Ogasawara M, Ozaki A, Ishiura H, Tsuji S, Minami N, Hayashi S, Noguchi S, Iida A, Nishino I; OPDM_LRP12 Study Group, Mori-Yoshimura M, Oya Y, Ono K, Shimizu T, Kawata A, Shimohama S, Toyooka K, Endo K, Toru S, Sasaki O, Isahaya K, Takahashi MP, Iwasa K, Kira JI, Yamamoto T, Kawamoto M, Hamano T, Sugie K, Eura N, Shiota T, Koide M, Sekiya K, Kishi H, Hideyama T, Kawai S, Yanagimoto S, Sato H, Arahata H, Murayama S, Saito K, Hara H, Kanda T, Yaguchi H, Imai N, Kawagashira Y, Sanada M, Obara K, Kaido M, Furuta M, Kurashige T, Hara W, Kuzume D, Yamamoto M, Tsugawa J, Kishida H, Ishizuka N, Morimoto K, Tsuji Y, Tsuneyama A, Matsuno A, Sasaki R, Tamakoshi D, Abe E, Yamada S, Uzawa A
JAMA Neurol 2021 Jul 1;78(7):853-863. doi: 10.1001/jamaneurol.2021.1509. PMID: 34047774Free PMC Article
Yu J, Deng J, Guo X, Shan J, Luan X, Cao L, Zhao J, Yu M, Zhang W, Lv H, Xie Z, Meng L, Zheng Y, Zhao Y, Gang Q, Wang Q, Liu J, Zhu M, Zhou B, Li P, Liu Y, Wang Y, Yan C, Hong D, Yuan Y, Wang Z
Brain 2021 Jul 28;144(6):1819-1832. doi: 10.1093/brain/awab077. PMID: 33693509Free PMC Article

Diagnosis

Owusu R, Savarese M
Acta Myol 2023;42(4):123-128. Epub 2023 Dec 20 doi: 10.36185/2532-1900-394. PMID: 38406378Free PMC Article
Yang X, Zhang D, Shen S, Li P, Li M, Niu J, Ma D, Xu D, Li S, Guo X, Wang Z, Zhao Y, Ren H, Ling C, Wang Y, Fan Y, Shen J, Zhu Y, Wang D, Cui L, Chen L, Shi C, Dai Y
BMC Med Genomics 2023 Oct 20;16(1):253. doi: 10.1186/s12920-023-01586-9. PMID: 37864208Free PMC Article
Yu J, Deng J, Wang Z
Curr Opin Neurol 2022 Oct 1;35(5):637-644. Epub 2022 Aug 4 doi: 10.1097/WCO.0000000000001089. PMID: 35942670
Zhou ZD, Jankovic J, Ashizawa T, Tan EK
Nat Rev Neurol 2022 Mar;18(3):145-157. Epub 2022 Jan 12 doi: 10.1038/s41582-021-00612-7. PMID: 35022573
Lu X, Hong D
J Neural Transm (Vienna) 2021 Mar;128(3):295-303. Epub 2021 Feb 18 doi: 10.1007/s00702-021-02313-3. PMID: 33599827

Prognosis

Tang H, Xiong Y, Jiang K, Shen Y, Yu Y, Huang P, Zhu M, Li X, Zheng Y, Zhou M, Yu J, Deng J, Wang Z, Hong D, Qiu Y, Tan D
Muscle Nerve 2024 Oct;70(4):744-752. Epub 2024 Jul 23 doi: 10.1002/mus.28200. PMID: 39044557
Durmus H, Laval SH, Deymeer F, Parman Y, Kiyan E, Gokyigiti M, Ertekin C, Ercan I, Solakoglu S, Karcagi V, Straub V, Bushby K, Lochmüller H, Serdaroglu-Oflazer P
Neurology 2011 Jan 18;76(3):227-35. doi: 10.1212/WNL.0b013e318207b043. PMID: 21242490
van der Sluijs BM, ter Laak HJ, Scheffer H, van der Maarel SM, van Engelen BG
J Neurol Neurosurg Psychiatry 2004 Oct;75(10):1499-501. doi: 10.1136/jnnp.2003.025072. PMID: 15377709Free PMC Article

Clinical prediction guides

Gu X, Yu J, Jiao K, Deng J, Xia X, Qiao K, Yue D, Gao M, Zhao C, Dong J, Huang G, Shan J, Yan C, Di L, Da Y, Zhu W, Xi J, Wang Z
J Med Genet 2024 Mar 21;61(4):340-346. doi: 10.1136/jmg-2023-109345. PMID: 37923380
Kume K, Kurashige T, Muguruma K, Morino H, Tada Y, Kikumoto M, Miyamoto T, Akutsu SN, Matsuda Y, Matsuura S, Nakamori M, Nishiyama A, Izumi R, Niihori T, Ogasawara M, Eura N, Kato T, Yokomura M, Nakayama Y, Ito H, Nakamura M, Saito K, Riku Y, Iwasaki Y, Maruyama H, Aoki Y, Nishino I, Izumi Y, Aoki M, Kawakami H
Am J Hum Genet 2023 Jul 6;110(7):1086-1097. Epub 2023 Jun 19 doi: 10.1016/j.ajhg.2023.05.014. PMID: 37339631Free PMC Article
Shimizu T, Ishiura H, Hara M, Shibata S, Unuma A, Kubota A, Sakuishi K, Inoue K, Goto J, Takahashi Y, Shirota Y, Hamada M, Shimizu J, Tsuji S, Toda T
Muscle Nerve 2022 Dec;66(6):679-685. Epub 2022 Sep 27 doi: 10.1002/mus.27717. PMID: 36052448
Ishiura H, Tsuji S
Curr Opin Genet Dev 2020 Dec;65:176-185. Epub 2020 Aug 7 doi: 10.1016/j.gde.2020.05.029. PMID: 32777681
Satoyoshi E, Kinoshita M
Arch Neurol 1977 Feb;34(2):89-92. doi: 10.1001/archneur.1977.00500140043007. PMID: 836191

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