Mariana Ferreira Francisco, Miguel Raimundo, Ana Luísa Carvalho, João Pedro Marques
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Clinical-electroretinography mismatch in a patient with non-syndromic BBS1-associated retinal degeneration.
A male patient in his late thirties, homozygous for the M390R Bardet-Biedl Syndrome-1 (BBS1) variant, began follow-up in early adulthood, due to decreased visual acuity and photophobia. Fundus examination showed bilateral temporal wedge-shaped pallor of the optic disc and atrophic maculopathy, translating into central hypoautofluorescence on fundus autofluorescence. Even though clinical and multimodal imaging features suggested cone-dominant degeneration, electroretinography (ERG) showed reduced scotopic and photopic responses, with greater scotopic impairment. Despite a thorough systemic evaluation and long-term follow-up, no other features of BBS were found. To our knowledge, this is the first documented case of non-syndromic BBS1-associated disease with a rod-cone dystrophy phenotype on ERG and a clinical presentation (symptoms and imaging features) of cone-dominant disease. The discrepancy between clinical/imaging and ERG findings reflects the complexity of BBS1-related disease and highlights the crucial role of genetic testing and comprehensive phenotyping in inherited retinal disorders.
期刊介绍:
BMJ Case Reports is an important educational resource offering a high volume of cases in all disciplines so that healthcare professionals, researchers and others can easily find clinically important information on common and rare conditions. All articles are peer reviewed and copy edited before publication. BMJ Case Reports is not an edition or supplement of the BMJ.