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Riboflavin transporter deficiency

MedGen UID:
1634394
Concept ID:
C4551777
Disease or Syndrome
Synonym: Brown-Vialetto-Van Laere syndrome
SNOMED CT: Progressive bulbar palsy with sensorineural deafness (699866005); Brown-Vialetto-Van Laere syndrome (699866005); Pontobulbar palsy with deafness (699866005); Riboflavin transporter deficiency (699866005)
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).
 
Monarch Initiative: MONDO:0008891
OMIM® Phenotypic series: PS211530
Orphanet: ORPHA97229

Definition

A rare, genetic motor neuron disease characterized by a peripheral and cranial neuropathy, neuronal loss in anterior horns and atrophy of spinal sensory tracts, causing muscle weakness, sensory loss, diaphragmatic paralysis and respiratory insufficiency, and multiple cranial nerve deficits such as sensorineural hearing loss, bulbar symptoms, and loss of vision due to optic atrophy. Depending on the transporter affected, Riboflavin transporter deficiency 2 (RFVT2) and Riboflavin transporter deficiency 3 (RFVT3) are distinguished. [from ORDO]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVRiboflavin transporter deficiency

Professional guidelines

PubMed

Bosch AM, Stroek K, Abeling NG, Waterham HR, Ijlst L, Wanders RJ
Orphanet J Rare Dis 2012 Oct 29;7:83. doi: 10.1186/1750-1172-7-83. PMID: 23107375Free PMC Article
Horvath R
J Inherit Metab Dis 2012 Jul;35(4):679-87. Epub 2012 Jan 10 doi: 10.1007/s10545-011-9434-1. PMID: 22231380

Recent clinical studies

Etiology

Jaeger B, Hoytema van Konijnenburg E, Groenveld MA, Langeveld M, Wolf NI, Bosch AM
Orphanet J Rare Dis 2024 Nov 1;19(1):410. doi: 10.1186/s13023-024-03428-y. PMID: 39487500Free PMC Article
Plantone D, Pardini M, Rinaldi G
Clin Drug Investig 2021 Jun;41(6):513-527. Epub 2021 Apr 22 doi: 10.1007/s40261-021-01038-1. PMID: 33886098
Console L, Tolomeo M, Colella M, Barile M, Indiveri C
Int J Mol Sci 2019 Sep 8;20(18) doi: 10.3390/ijms20184416. PMID: 31500345Free PMC Article
Cornett KMD, Menezes MP, Bray P, Halaki M, Burns J
J Peripher Nerv Syst 2018 Mar;23(1):29-35. Epub 2017 Dec 11 doi: 10.1111/jns.12245. PMID: 29168276
Menezes MP, O'Brien K, Hill M, Webster R, Antony J, Ouvrier R, Birman C, Gardner-Berry K
Dev Med Child Neurol 2016 Aug;58(8):848-54. Epub 2016 Feb 25 doi: 10.1111/dmcn.13084. PMID: 26918385

Diagnosis

Bulas S, Bedoukian EC, O'Neil EC, Krantz ID, Yum SW, Liu GT, Aleman TS
J Neuroophthalmol 2023 Mar 1;43(1):110-115. Epub 2022 Aug 2 doi: 10.1097/WNO.0000000000001678. PMID: 35921603
do Amaral MSA, Massuda ET, Mitikami Fenólio GH, Barbosa Reis ACM, Angelo Hyppolito M
J Int Adv Otol 2022 Mar;18(2):192-195. doi: 10.5152/iao.2022.21159. PMID: 35418370Free PMC Article
Kranthi P, Garuda BR, Gopi S, Kumar TS
Neurol India 2020 Sep-Oct;68(5):1217-1219. doi: 10.4103/0028-3886.299175. PMID: 33109881
Kaphan E, Bou Ali H, Gastaldi M, Acquaviva C, Vianey-Saban C, Rouzier C, Fragaki K, Bannwarth S, Paquis-Flucklinger V, Romero N, Behin A, Lombès A, Jardel C, Rigal O, Laforêt P
Rev Neurol (Paris) 2018 Dec;174(10):731-735. Epub 2018 Oct 11 doi: 10.1016/j.neurol.2018.03.014. PMID: 30318261
Allison T, Roncero I, Forsyth R, Coffman K, Pichon JL
J Child Neurol 2017 May;32(6):528-532. Epub 2017 Jan 24 doi: 10.1177/0883073816689517. PMID: 28116953

Therapy

Fennessy JR, Cornett KMD, Burns J, Menezes MP
J Peripher Nerv Syst 2023 Sep;28(3):308-316. Epub 2023 Aug 24 doi: 10.1111/jns.12587. PMID: 37537696
Elks N, Wilmshurst JM, Raga SV
Pediatr Neurol 2023 Jul;144:16-18. Epub 2023 Apr 7 doi: 10.1016/j.pediatrneurol.2023.04.004. PMID: 37116404
Bulas S, Bedoukian EC, O'Neil EC, Krantz ID, Yum SW, Liu GT, Aleman TS
J Neuroophthalmol 2023 Mar 1;43(1):110-115. Epub 2022 Aug 2 doi: 10.1097/WNO.0000000000001678. PMID: 35921603
Plantone D, Pardini M, Rinaldi G
Clin Drug Investig 2021 Jun;41(6):513-527. Epub 2021 Apr 22 doi: 10.1007/s40261-021-01038-1. PMID: 33886098
Carreau C, Lenglet T, Mosnier I, Lahlou G, Fargeot G, Weiss N, Demeret S, Salachas F, Veauville-Merllié A, Acquaviva C, Nadjar Y
Ann Clin Transl Neurol 2020 Feb;7(2):250-253. Epub 2020 Feb 5 doi: 10.1002/acn3.50977. PMID: 32022482Free PMC Article

Prognosis

Plantone D, Pardini M, Rinaldi G
Clin Drug Investig 2021 Jun;41(6):513-527. Epub 2021 Apr 22 doi: 10.1007/s40261-021-01038-1. PMID: 33886098
Gayathri S, Gowda VK, Udhayabanu T, O'Callaghan B, Efthymiou S, Varalakshmi P, Benakappa N, Houlden H, Ashokkumar B
Eur J Neurol 2021 Mar;28(3):945-954. Epub 2021 Jan 5 doi: 10.1111/ene.14682. PMID: 33325104
Kranthi P, Garuda BR, Gopi S, Kumar TS
Neurol India 2020 Sep-Oct;68(5):1217-1219. doi: 10.4103/0028-3886.299175. PMID: 33109881
Jaeger B, Bosch AM
J Inherit Metab Dis 2016 Jul;39(4):559-64. Epub 2016 Mar 14 doi: 10.1007/s10545-016-9924-2. PMID: 26973221Free PMC Article
Menezes MP, O'Brien K, Hill M, Webster R, Antony J, Ouvrier R, Birman C, Gardner-Berry K
Dev Med Child Neurol 2016 Aug;58(8):848-54. Epub 2016 Feb 25 doi: 10.1111/dmcn.13084. PMID: 26918385

Clinical prediction guides

Fennessy JR, Donlevy GA, McKay MJ, Burns J, Cornett KMD, Menezes MP
J Peripher Nerv Syst 2024 Jun;29(2):185-192. Epub 2024 Mar 6 doi: 10.1111/jns.12619. PMID: 38445790
Nair AV, Kandagaddala M, Sivadasan A, Prabhakar AT, Nair S, Mathew V, Aaron S, Alexander M
J Clin Neuromuscul Dis 2024 Mar 1;25(3):122-131. doi: 10.1097/CND.0000000000000465. PMID: 38441928
Gayathri S, Gowda VK, Udhayabanu T, O'Callaghan B, Efthymiou S, Varalakshmi P, Benakappa N, Houlden H, Ashokkumar B
Eur J Neurol 2021 Mar;28(3):945-954. Epub 2021 Jan 5 doi: 10.1111/ene.14682. PMID: 33325104
Amir F, Atzinger C, Massey K, Greinwald J, Hunter LL, Ulm E, Kettler M
J Child Neurol 2020 Mar;35(4):283-290. Epub 2019 Dec 23 doi: 10.1177/0883073819893159. PMID: 31868069
Mandarakas MR, Menezes MP, Rose KJ, Shy R, Eichinger K, Foscan M, Estilow T, Kennedy R, Herbert K, Bray P, Refshauge K, Ryan MM, Yiu EM, Farrar M, Sampaio H, Moroni I, Pagliano E, Pareyson D, Yum SW, Herrmann DN, Acsadi G, Shy ME, Burns J, Sanmaneechai O
Brain 2018 Dec 1;141(12):3319-3330. doi: 10.1093/brain/awy280. PMID: 30476010Free PMC Article

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