Primary coenzyme Q10 deficiency: an update

D Mantle, L Millichap, J Castro-Marrero, IP Hargreaves - Antioxidants, 2023 - mdpi.com
Coenzyme Q10 (CoQ10) has a number of vital functions in all cells, both mitochondrial and
extra-mitochondrial. In addition to its key role in mitochondrial oxidative phosphorylation …

Childhood nephrotic syndrome

M Vivarelli, K Gibson, A Sinha, O Boyer - The Lancet, 2023 - thelancet.com
Idiopathic nephrotic syndrome is the most common glomerular disease in children.
Corticosteroids are the cornerstone of its treatment, and steroid response is the main …

Increased risk of kidney failure in patients with genetic kidney disorders

MD Elliott, N Vena, M Marasa, E Cocchi, S Bheda… - The Journal of clinical …, 2024 - jci.org
BACKGROUND It is unknown whether the risk of kidney disease progression and failure
differs between patients with and without genetic kidney disorders. METHODS Three cohorts …

[HTML][HTML] State of the art in childhood nephrotic syndrome: concrete discoveries and unmet needs

F Vincenti, A Angeletti, GM Ghiggeri - Frontiers in Immunology, 2023 - ncbi.nlm.nih.gov
Nephrotic syndrome (NS) is a clinical entity characterized by proteinuria, hypoalbuminemia,
and peripheral edema. NS affects about 2–7 per 100,000 children aged below 18 years old …

[HTML][HTML] Variation of the clinical spectrum and genotype-phenotype associations in Coenzyme Q10 deficiency associated glomerulopathy

S Drovandi, BS Lipska-Ziętkiewicz, F Ozaltin, F Emma… - Kidney International, 2022 - Elsevier
Primary Coenzyme Q10 deficiency is a rare mitochondriopathy with a wide spectrum of
organ involvement, including steroid-resistant nephrotic syndrome mainly associated with …

Mechanisms of podocyte injury in genetic kidney disease

N Mann, H Sun, AJ Majmundar - Pediatric Nephrology, 2024 - Springer
Glomerular diseases are a leading cause of chronic kidney disease worldwide. Both
acquired and hereditary glomerulopathies frequently share a common final disease …

Gene therapy for mitochondrial disorders

N Keshavan, M Minczuk, C Viscomi… - Journal of Inherited …, 2024 - Wiley Online Library
In this review, we detail the current state of application of gene therapy to primary
mitochondrial disorders (PMDs). Recombinant adeno‐associated virus‐based (rAAV) gene …

Idiopathic collapsing glomerulopathy is associated with APOL1 high-risk genotypes or Mendelian variants in most affected individuals in a highly admixed population

PD Neves, A Watanabe, EH Watanabe, AM Narcizo… - Kidney International, 2024 - Elsevier
Collapsing glomerulopathy (CG) is most often associated with fast progression to kidney
failure with an incidence apparently higher in Brazil than in other countries. However, the …

Syndromic Retinitis Pigmentosa

JS Karuntu, H Almushattat, AS Plomp… - Progress in Retinal and …, 2024 - Elsevier
Retinitis pigmentosa (RP) is a progressive inherited retinal dystrophy, characterized by the
degeneration of photoreceptors, presenting as a rod-cone dystrophy. Approximately 20-30 …

Clinical Features, Biochemistry, Imaging, and Treatment Response in a Single-Center Cohort With Coenzyme Q10 Biosynthesis Disorders

A Wahedi, S Sudhakar, A Lam, JIR Ciancio… - Neurology …, 2024 - neurology.org
Background and Objectives Disorders of coenzyme Q10 (CoQ10) biosynthesis comprise a
group of 11 clinically and genetically heterogeneous rare primary mitochondrial diseases …