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Published Work

The Fruits of Our Labor

Publications: Publications

2019

BMC Nephrol.

CTNS mRNA molecular analysis revealed a novel mutation in a child with infantile nephropathic cystinosis: a case report.

BMC Nephrol. 2019 Oct 31;20(1):400. doi: 10.1186/s12882-019-1589-2.

Publications: Publications

2019

Pediatr Nephrol. 

The European Society for Paediatric Nephrology study of pediatric renal care in Europe: comparative analysis 1998-2017. Pediatr Nephrol. 2020 Jan;35(1):103-111. doi: 10.1007/s00467-019-04378-5. Epub 2019 Oct 29.

Publications: Publications

2019

Ophthalmol Ther.

Ophthalmic Outcome in a Belgian Cohort of Cystinosis Patients Treated with a Compounded Preparation of Cysteamine Eye Drops: Retrospective Analysis. Ophthalmol Ther. 2019 Dec;8(4):623-633. doi: 10.1007/s40123-019-00216-x. Epub 2019 Sep 27.

Publications: Publications

2019

BMJ Open

Prevention of relapses with levamisole as adjuvant therapy in children with a first episode of idiopathic nephrotic syndrome: study protocol for a double blind, randomised placebo-controlled trial (the LEARNS study). BMJ Open. 2019 Aug 1;9(8):e027011. doi: 10.1136/bmjopen-2018-027011.

Publications: Publications

2019

Kidney Int Rep

APOL1 Risk Genotypes Are Associated With Early Kidney Damage in Children in Sub-Saharan Africa. Kidney Int Rep. 2019 Apr 11;4(7):930-938. doi: 10.1016/j.ekir.2019.04.002. eCollection 2019 Jul.

Publications: Publications

2019

J Am Soc Nephrol

Genetic Identification of Two Novel Loci Associated with Steroid-Sensitive Nephrotic Syndrome. J Am Soc Nephrol. 2019 Aug;30(8):1375-1384. doi: 10.1681/ASN.2018101054. Epub 2019 Jul 1.

Publications: Publications

2019

J Invest Dermatol

Enhanced Intrinsic Skin Aging in Nephropathic Cystinosis Assessed by High-Definition Optical Coherence Tomography. J Invest Dermatol. 2019 Apr 22. pii: S0022-202X(19)31491-5. doi: 10.1016/j.jid.2019.03.1153. [Epub ahead of print]

Publications: Publications

2019

Nephron

Molecular Basis of Cystinosis: Geographic Distribution, Functional Consequences of Mutations in the CTNS Gene, and Potential for Repair. Nephron. 2019;141(2):133-146. doi: 10.1159/000495270. Epub 2018 Dec 14

Publications: Publications

2018

Sci Rep

Activation of Calcium-Sensing Receptor increases intracellular calcium and decreases cAMP and mTOR in PKD1 deficient cells. Sci Rep. 2018 Apr 9;8(1):5704. doi: 10.1038/s41598-018-23732-5.

Publications: Publications

2018

Matrix Biol.

Connective tissue growth factor (CTGF) from basics to clinics. Matrix Biol. 2018 Aug;68-69:44-66. doi: 10.1016/j.matbio.2018.03.007. Epub 2018 Mar 21.

Publications: Publications

2018

Arch Dis Child Fetal Neonatal Ed.

Ibuprofen exposure in early neonatal life does not affect renal function in young adolescence. Arch Dis Child Fetal Neonatal Ed. 2018 Mar;103(2):F107-F111. doi: 10.1136/archdischild-2017-312922. Epub 2017 Jun 14.

Publications: Publications

2018

Cells

Genetic Renal Diseases: The Emerging Role of Zebrafish Models. Cells. 2018 Sep 1;7(9). pii: E130. doi: 10.3390/cells7090130.

Publications: Publications

2018

Eur J Pediatr

Renal Replacement Therapy in children with severe developmental disability: guiding questions for decision-making. Eur J Pediatr. 2018 Dec;177(12):1735-1743. doi: 10.1007/s00431-018-3238-3. Epub 2018 Sep 7.

Publications: Publications

2018

Am J Transplant.

Allogeneic HSCT transfers wild-type cystinosin to nonhematological epithelial cells in cystinosis: First human report. Am J Transplant. 2018 Nov;18(11):2823-2828. doi: 10.1111/ajt.15029. Epub 2018 Aug 21.

Publications: Publications

2018

JIMD Rep

Effect of Storage Conditions on Stability of Ophthalmological Compounded Cysteamine Eye Drops. JIMD Rep. 2018;42:47-51. doi: 10.1007/8904_2017_77. Epub 2017 Dec 7.

Publications: Publications

2017

Hypertension

Simplified Thresholds for Pediatric (Pre)hypertension: One Approach to Improve Recognition and Outcome. Hypertension. 2017 Apr;69(4):587-588. doi: 10.1161/HYPERTENSIONAHA.116.08883. Epub 2017 Feb 21.

Publications: Publications

2017

Hypertension

Does Extremely Low Birth Weight Predispose to Low-Renin Hypertension? Hypertension. 2017 Mar;69(3):443-449. doi: 10.1161/HYPERTENSIONAHA.116.08643. Epub 2017 Jan 23.

Publications: Publications

2017

Ophthalmol Ther

Clinical Practice: A Proposed Standardized Ophthalmological Assessment for Patients with Cystinosis. Ophthalmol Ther. 2017 Jun;6(1):93-104. doi: 10.1007/s40123-017-0089-3. Epub 2017 May 5.

Publications: Publications

2017

PLoS One

Catch-up growth in the first two years of life in Extremely Low Birth Weight (ELBW) infants is associated with lower body fat in young adolescence. PLoS One. 2017 Mar 9;12(3):e0173349. doi: 10.1371/journal.pone.0173349. eCollection 2017.

Publications: Publications

2017

Curr Pharm Des

A Human Proximal Tubular Epithelial Cell Model to Explore a Knowledge Gap on Neonatal Drug Disposition. Curr Pharm Des. 2017;23(38):5911-5918. doi: 10.2174/1381612823666171009143146.

Publications: Publications

2017

PLoS One

Pituitary adenylate cyclase-activating polypeptide (PACAP) in zebrafish models of nephrotic syndrome. PLoS One. 2017 Jul 31;12(7):e0182100. doi: 10.1371/journal.pone.0182100. eCollection 2017.

Publications: Publications

2016

Sci Rep

TRPV1 dysfunction in cystinosis patients harboring the homozygous 57 kb deletion. Sci Rep. 2016; 6: 35395.

Published online 2016 Oct 13. doi: 10.1038/srep35395

Publications: Publications

2016

Crit Care Med

Vascular Endothelial Growth Factor Up-regulation in Human Amniotic Fluid Stem Cell Enhances Nephroprotection After Ischemia-Reperfusion Injury in the Rat. Crit Care Med. 2017 Jan;45(1):e86-e96.

Publications: Publications

2016

N Engl J Med

Polyhydramnios, Transient Antenatal Bartter's Syndrome, and MAGED2 Mutations. N Engl J Med. 2016 May 12;374(19):1853-63. doi: 10.1056/NEJMoa1507629. Epub 2016 Apr 27.

Publications: Publications

2016

Pediatr Nephrol

Platelet abnormalities in nephrotic syndrome. Pediatr Nephrol. 2016 Aug;31(8):1267-79. doi: 10.1007/s00467-015-3173-8. Epub 2015 Aug 13.

Publications: Publications

2016

Kidney Int

Controversies and research agenda in nephropathic cystinosis: conclusions from a "Kidney Disease: Improving Global Outcomes" (KDIGO) Controversies Conference. Kidney Int. 2016 Jun;89(6):1192-203. doi: 10.1016/j.kint.2016.01.033.

Publications: Publications

2016

J Am Soc Nephrol

Autosomal-Recessive Mutations in SLC34A1 Encoding Sodium-Phosphate Cotransporter 2A Cause Idiopathic Infantile Hypercalcemia. J Am Soc Nephrol. 2016 Feb;27(2):604-14. doi: 10.1681/ASN.2014101025. Epub 2015 Jun 5.

Publications: Publications

2016

Nat Cell Biol

Autophagosome-lysosome fusion triggers a lysosomal response mediated by TLR9 and controlled by OCRL. Nat Cell Biol. 2016 Aug;18(8):839-850. doi: 10.1038/ncb3386. Epub 2016 Jul 11.

Publications: Publications

2015

Pediatr Nephrol

Urinary excretion of polyols and sugars in children with chronic kidney disease. Pediatr Nephrol. 2015 Sep;30(9):1537-40. doi: 10.1007/s00467-015-3135-1. Epub 2015 Jun 9.

Publications: Publications

2016

Cystinosis: a new perspective. Acta Clin Belg. 2016 Jun;71(3):131-7. doi: 10.1179/2295333714Y.0000000113.

 Acta Clin Belg

Publications: Publications

2015

J Thromb Haemost

Pituitary adenylate cyclase-activating polypeptide deficiency associated with increased platelet count and aggregability in nephrotic syndrome. J Thromb Haemost. 2015 May;13(5):755-67. doi: 10.1111/jth.12891. Epub 2015 Apr 13.

Publications: Publications

2015

Am J Physiol Renal Physiol

Conditionally immortalized human proximal tubular epithelial cells isolated from the urine of a healthy subject express functional calcium-sensing receptor. Am J Physiol Renal Physiol. 2015 Jun 1;308(11):F1200-6. doi: 10.1152/ajprenal.00352.2014. Epub 2015 Feb 4.

Publications: Publications

2014

JIMD Rep

Carnitine Profile and Effect of Suppletion in Children with Renal Fanconi Syndrome due to Cystinosis. JIMD Rep. 2014;16:25-30. doi: 10.1007/8904_2014_312. Epub 2014 May 6.

Publications: Publications

2014

Nephrol Dial Transplant

Nephropathic cystinosis: an international consensus document. Nephrol Dial Transplant. 2014 Sep;29 Suppl 4:iv87-94. doi: 10.1093/ndt/gfu090.

Publications: Publications

2015

 PLoS One

Amniotic Fluid Derived Stem Cells with a Renal Progenitor Phenotype Inhibit Interstitial Fibrosis in Renal Ischemia and Reperfusion Injury in Rats. PLoS One. 2015 Aug 21;10(8):e0136145. doi: 10.1371/journal.pone.0136145. eCollection 2015.

Publications: Publications

2014

J Clin Endocrinol Metab

Switch in FGFR3 and -4 expression profile during human renal development may account for transient hypercalcemia in patients with Sotos syndrome due to 5q35 microdeletions. J Clin Endocrinol Metab. 2014 Jul;99(7):E1361-7. doi: 10.1210/jc.2014-1123. Epub 2014 Mar 26.

Publications: Publications

2014

Eur J Hum Genet

Clinical utility gene card for: Dent disease (Dent-1 and Dent-2). Eur J Hum Genet. 2014 Nov;22(11). doi: 10.1038/ejhg.2014.33. Epub 2014 Mar 12.

Publications: Publications

2014

J Am Soc Nephrol

Time course of pathogenic and adaptation mechanisms in cystinotic mouse kidneys. J Am Soc Nephrol. 2014 Jun;25(6):1256-69. doi: 10.1681/ASN.2013060598. Epub 2014 Feb 13.

Publications: Publications

2014

JIMD Rep

Mutational Spectrum of the CTNS Gene in Egyptian Patients with Nephropathic Cystinosis. JIMD Rep. 2014;14:87-97. doi: 10.1007/8904_2013_288. Epub 2014 Jan 25.

Publications: Publications

2014

Pediatrics.

An unusual presentation of Denys-Drash syndrome due to bigenic disease. Pediatrics. 2014 Jan;133(1):e252-6. doi: 10.1542/peds.2013-1524. Epub 2013 Dec 30.

Publications: Publications

2014

Eur J Hum Genet

Clinical utility gene card for: cystinosis. Eur J Hum Genet. 2014 May;22(5). doi: 10.1038/ejhg.2013.204. Epub 2013 Sep 18.

Publications: Publications

2013

J Pediatr Gastroenterol Nutr

Another cause of hyperglyceroluria: aquaporin 7 gene mutation. J Pediatr Gastroenterol Nutr. 2013 Sep;57(3):e19. doi: 10.1097/MPG.0b013e31829a4ff3.

Publications: Publications

2013

Proc Natl Acad Sci U S A

Receptor-mediated endocytosis and endosomal acidification is impaired in proximal tubule epithelial cells of Dent disease patients. Proc Natl Acad Sci U S A. 2013 Apr 23;110(17):7014-9. doi: 10.1073/pnas.1302063110. Epub 2013 Apr 9.

Publications: Publications

2013

J Inherit Metab Dis

Increased human dermal microvascular endothelial cell survival induced by cysteamine. J Inherit Metab Dis. 2013 Nov;36(6):1073-7. doi: 10.1007/s10545-013-9588-0. Epub 2013 Jan 31.

Publications: Publications

2012

PLoS One

Stem cell microvesicles transfer cystinosin to human cystinotic cells and reduce cystine accumulation in vitro. PLoS One. 2012;7(8):e42840. doi: 10.1371/journal.pone.0042840. Epub 2012 Aug 13.

Publications: Publications

2012

Histochem Cell Biol

Distribution of cystinosin-LKG in human tissues. Histochem Cell Biol. 2012 Aug;138(2):351-63. doi: 10.1007/s00418-012-0958-8. Epub 2012 Apr 29.

Publications: Publications

2011

EMBO J.

OCRL controls trafficking through early endosomes via PtdIns4,5P₂-dependent regulation of endosomal actin. EMBO J. 2011 Oct 4;30(24):4970-85. doi: 10.1038/emboj.2011.354.

Publications: Publications

2011

Mol Genet Metab

Elevated concentrations of sedoheptulose in bloodspots of patients with cystinosis caused by the 57-kb deletion: implications for diagnostics and neonatal screening. Mol Genet Metab. 2011 Mar;102(3):339-42. doi: 10.1016/j.ymgme.2010.12.002. Epub 2010 Dec 13.

Publications: Publications

2011

 Clin Chem

Necessity of fractionated urine collection for monitoring patients with cystinuria. Clin Chem. 2011 May;57(5):780-1. doi: 10.1373/clinchem.2010.161547. Epub 2011 Mar 15.

Publications: Publications

2010

Growth retardation in children with cystinosis. Minerva Pediatr. 2010 Jun;62(3):307-14.

Minerva Pediatr.

Publications: Publications

2010

Pediatr Nephrol

Analysis of CTNS gene transcripts in nephropathic cystinosis. Pediatr Nephrol. 2010 Jul;25(7):1263-7. doi: 10.1007/s00467-010-1502-5. Epub 2010 Mar 30.

Publications: Publications

2008

Nephrol Dial Transplant

Evaluation of the proximal tubular function in hereditary renal Fanconi syndrome. Nephrol Dial Transplant. 2008 Sep;23(9):2719-22. doi: 10.1093/ndt/gfn373. Epub 2008 Jul 4.

Publications: Publications

2008

Clin J Am Soc Nephrol

Renal phenotype in Lowe Syndrome: a selective proximal tubular dysfunction. Clin J Am Soc Nephrol. 2008 Sep;3(5):1430-6. doi: 10.2215/CJN.00520108. Epub 2008 May 14.

Publications: Publications

2008

Am J Physiol Renal Physiol

Identification and subcellular localization of a new cystinosin isoform. Am J Physiol Renal Physiol. 2008 May;294(5):F1101-8. doi: 10.1152/ajprenal.00413.2007. Epub 2008 Mar 12.

Publications: Publications

2008

Hum Mutat

Sedoheptulokinase deficiency due to a 57-kb deletion in cystinosis patients causes urinary accumulation of sedoheptulose: elucidation of the CARKL gene. Hum Mutat. 2008 Apr;29(4):532-6. doi: 10.1002/humu.20685.

Publications: Publications

2017

JIMD Rep

First Successful Conception Induced by a Male Cystinosis Patient. JIMD Rep. 2018;38:1-6. doi: 10.1007/8904_2017_19. Epub 2017 Apr 13.

2017

Endocrine Complications of Cystinosis. J Pediatr. 2017 Apr;183S:S5-S8. doi: 10.1016/j.jpeds.2016.12.049.

J Pediatr.

2017

Sci Rep

Cystinosis (ctns) zebrafish mutant shows pronephric glomerular and tubular dysfunction. Sci Rep. 2017 Feb 15;7:42583. doi: 10.1038/srep42583.

2017

Curr Opin Pediatr

Nephropathic cystinosis: an update. Curr Opin Pediatr. 2017 Apr;29(2):168-178. doi: 10.1097/MOP.0000000000000462.

2017

Carbohydr Res

Evaluation of carbohydrate-cysteamine thiazolidines as pro-drugs for the treatment of cystinosis. Carbohydr Res. 2017 Feb 1;439:9-15. doi: 10.1016/j.carres.2016.12.003. Epub 2016 Dec 18.

2017

Br J Clin Pharmacol

Tacrolimus dose requirements in paediatric renal allograft recipients are characterized by a biphasic course determined by age and bone maturation. Br J Clin Pharmacol. 2017 Apr;83(4):863-874. doi: 10.1111/bcp.13174. Epub 2016 Dec 13.

2016

Cell Calcium

Ca(2+) signalling in human proximal tubular epithelial cells deficient for cystinosin. Cell Calcium. 2016 Oct;60(4):282-7. doi: 10.1016/j.ceca.2016.07.001. Epub 2016 Jul 5.

2016

Cystinosis: a review. Orphanet J Rare Dis. 2016 Apr 22;11:47. doi: 10.1186/s13023-016-0426-y.

Orphanet J Rare Dis

2016

Kidney Int.

Cystinosin deficiency causes podocyte damage and loss associated with increased cell motility. Kidney Int. 2016 May;89(5):1037-1048. doi: 10.1016/j.kint.2016.01.013. Epub 2016 Mar 11.

2016

Nephron

Distribution and Function of PACAP and Its Receptors in the Healthy and Nephrotic kidney. Nephron. 2016;132(4):301-11. doi: 10.1159/000445035. Epub 2016 Apr 7.

2016

J Am Soc Nephrol.

Urine of Preterm Neonates as a Novel Source of Kidney Progenitor Cells. J Am Soc Nephrol. 2016 Sep;27(9):2762-70. doi: 10.1681/ASN.2015060664. Epub 2016 Mar 3.

2016

J Inherit Metab Dis

Altered mTOR signalling in nephropathic cystinosis. J Inherit Metab Dis. 2016 May;39(3):457-464. doi: 10.1007/s10545-016-9919-z. Epub 2016 Feb 24.

2016

Enzyme Res

Immunomodulatory Effects of Chitotriosidase Enzyme. Enzyme Res. 2016;2016:2682680. doi: 10.1155/2016/2682680. Epub 2016 Jan 3.

2016

Pediatr Nephrol.

Autophagy in renal diseases. Pediatr Nephrol. 2016 May;31(5):737-52. doi: 10.1007/s00467-015-3134-2. Epub 2015 Jul 4.

2015

Stem Cells Int

Human Urine as a Noninvasive Source of Kidney Cells. Stem Cells Int. 2015;2015:362562. doi: 10.1155/2015/362562. Epub 2015 May 18.

2015

Simplified screening criteria for HNF1B analysis. Kidney Int. 2015 Jun;87(6):1258-9. doi: 10.1038/ki.2015.49.

Kidney Int

2015

Pediatr Nephrol.

Management dilemmas in pediatric nephrology: Cystinosis. Pediatr Nephrol. 2015 Aug;30(8):1349-60. doi: 10.1007/s00467-015-3117-3. Epub 2015 May 9.

2015

Early Hum Dev

Neonatal creatinemia trends as biomarker of subsequent cognitive outcome in extremely low birth weight neonates. Early Hum Dev. 2015 Jun;91(6):367-72. doi: 10.1016/j.earlhumdev.2015.03.008. Epub 2015 Apr 25.

2015

PLoS One

Endo-lysosomal dysfunction in human proximal tubular epithelial cells deficient for lysosomal cystine transporter cystinosin. PLoS One. 2015 Mar 26;10(3):e0120998. doi: 10.1371/journal.pone.0120998. eCollection 2015.

2015

Neuromuscul Disord

Renal function in children and adolescents with Duchenne muscular dystrophy. Neuromuscul Disord. 2015 May;25(5):381-7. doi: 10.1016/j.nmd.2015.01.005. Epub 2015 Jan 19.

2015

Mol Pharm

The functional implications of common genetic variation in CYP3A5 and ABCB1 in human proximal tubule cells. Mol Pharm. 2015 Mar 2;12(3):758-68. doi: 10.1021/mp500590s. Epub 2015 Jan 29.

2015

Cystinosis: a new perspective. Acta Clin Belg. 2016 Jun;71(3):131-7. doi: 10.1179/2295333714Y.0000000113.

Acta Clin Belg

2015

Nephrol Dial Transplant

Criteria for HNF1B analysis in patients with congenital abnormalities of kidney and urinary tract. Nephrol Dial Transplant. 2015 May;30(5):835-42. doi: 10.1093/ndt/gfu370. Epub 2014 Dec 13.

2014

Orphanet J Rare Dis

Clinical utility of chitotriosidase enzyme activity in nephropathic cystinosis. Orphanet J Rare Dis. 2014 Nov 19;9:155. doi: 10.1186/s13023-014-0155-z.

2014

Pediatr Endocrinol Rev

Cystinosis: clinical presentation, pathogenesis and treatment. Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:176-84.

2014

Int J Nephrol Renovasc Dis

Improving the prognosis of nephropathic cystinosis. Int J Nephrol Renovasc Dis. 2014 Jul 17;7:297-302. doi: 10.2147/IJNRD.S37603. eCollection 2014.

2014

Pflugers Arch

Polycystin-1 but not polycystin-2 deficiency causes upregulation of the mTOR pathway and can be synergistically targeted with rapamycin and metformin. Pflugers Arch. 2014 Aug;466(8):1591-604. doi: 10.1007/s00424-013-1394-x. Epub 2013 Nov 6.

2013

Int J Pharm

From gut to kidney: transporting and metabolizing calcineurin-inhibitors in solid organ transplantation. Int J Pharm. 2013 Aug 16;452(1-2):14-35. doi: 10.1016/j.ijpharm.2013.05.033. Epub 2013 May 24.

2013

J Pediatr.

Copper deficiency in patients with cystinosis with cysteamine toxicity. J Pediatr. 2013 Sep;163(3):754-60. doi: 10.1016/j.jpeds.2013.03.078. Epub 2013 May 4.

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2013

Drug Discov Today

Cysteamine: an old drug with new potential. Drug Discov Today. 2013 Aug;18(15-16):785-92. doi: 10.1016/j.drudis.2013.02.003. Epub 2013 Feb 14.

2012

Mol Genet Metab

Halitosis in cystinosis patients after administration of immediate-release cysteamine bitartrate compared to delayed-release cysteamine bitartrate. Mol Genet Metab. 2012 Sep;107(1-2):234-6. doi: 10.1016/j.ymgme.2012.06.017. Epub 2012 Jul 6.

2012

Pediatr Nephrol

Detailed studies of growth hormone secretion in cystinosis patients. Pediatr Nephrol. 2012 Nov;27(11):2123-2127. doi: 10.1007/s00467-012-2213-x. Epub 2012 Jun 5.

2012

Fertil Steril

Studying nonobstructive azoospermia in cystinosis: histologic examination of testes and epididymis and sperm analysis in a Ctns⁻/⁻ mouse model. Fertil Steril. 2012 Jul;98(1):162-5. doi: 10.1016/j.fertnstert.2012.03.050. Epub 2012 May 10.

2011

Clin J Am Soc Nephrol

Improvement in the renal prognosis in nephropathic cystinosis. Clin J Am Soc Nephrol. 2011 Oct;6(10):2485-91. doi: 10.2215/CJN.02000311. Epub 2011 Aug 25.

2011

J Pediatr

Cysteamine toxicity in patients with cystinosis. J Pediatr. 2011 Dec;159(6):1004-11. doi: 10.1016/j.jpeds.2011.05.057. Epub 2011 Jul 23.

2011

Pediatrics

Apparent mineralocorticoid excess: time of manifestation and complications despite treatment. Pediatrics. 2011 Jun;127(6):e1610-4. doi: 10.1542/peds.2010-1928. Epub 2011 May 2.

2011

Biochim Biophys Acta

Cysteamine restores glutathione redox status in cultured cystinotic proximal tubular epithelial cells. Biochim Biophys Acta. 2011 Jun;1812(6):643-51. doi: 10.1016/j.bbadis.2011.02.010. Epub 2011 Feb 28.

2011

J Inherit Metab Dis

Non-invasive measurements of atherosclerosis in adult cystinosis patients. J Inherit Metab Dis. 2011 Jun;34(3):811-8. doi: 10.1007/s10545-011-9281-0. Epub 2011 Feb 9.

2011

Pharmaceutics

Role of p-glycoprotein expression and function in cystinotic renal proximal tubular cells. Pharmaceutics. 2011 Oct 27;3(4):782-92. doi: 10.3390/pharmaceutics3040782.

2011

Pediatr Nephro

Pharmacokinetics of cysteamine in a cystinosis patient treated with hemodialysis. Pediatr Nephrol. 2011 Apr;26(4):639-40. doi: 10.1007/s00467-010-1680-1. Epub 2010 Oct 24.

2010

Am J Physiol Renal Physiol.

The pathogenesis of cystinosis: mechanisms beyond cystine accumulation. Am J Physiol Renal Physiol. 2010 Nov;299(5):F905-16. doi: 10.1152/ajprenal.00318.2010. Epub 2010 Sep 8.

2010

Pediatr Nephrol.

Cystinosis: practical tools for diagnosis and treatment. Pediatr Nephrol. 2011 Feb;26(2):205-15. doi: 10.1007/s00467-010-1627-6. Epub 2010 Aug 24.

2010

Cell Tissue Res.

Novel conditionally immortalized human proximal tubule cell line expressing functional influx and efflux transporters. Cell Tissue Res. 2010 Feb;339(2):449-57. doi: 10.1007/s00441-009-0882-y. Epub 2009 Nov 10.

2010

Fertil Steril

Fertility status in male cystinosis patients treated with cysteamine. Fertil Steril. 2010 Apr;93(6):1880-3. doi: 10.1016/j.fertnstert.2008.12.113. Epub 2009 Feb 12.

2008

Gitelman syndrome. Orphanet J Rare Dis. 2008 Jul 30;3:22. doi: 10.1186/1750-1172-3-22.

Orphanet J Rare Dis

2008

Pediatr Res

Mitochondrial complex V expression and activity in cystinotic fibroblasts. Pediatr Res. 2008 Nov;64(5):495-7. doi: 10.1203/PDR.0b013e318183fd67.

2008

Am J Kidney Dis

Urinary protein excretion pattern and renal expression of megalin and cubilin in nephropathic cystinosis. Am J Kidney Dis. 2008 Jun;51(6):893-903. doi: 10.1053/j.ajkd.2008.03.010. Epub 2008 May 2.

2008

Neurochem Res

The use of CDME in cystinosis research. Neurochem Res. 2008 Nov;33(11):2373-4. doi: 10.1007/s11064-008-9709-6. Epub 2008 Apr 22.

2007

Cystine dimethylester model of cystinosis: still reliable? Pediatr Res. 2007 Aug;62(2):151-5.

Pediatr Res

2007

Mol Genet Metab

The origin of halitosis in cystinotic patients due to cysteamine treatment. Mol Genet Metab. 2007 Jul;91(3):228-33. Epub 2007 May 21.

2006

Acta Paediatr.

Development of Fanconi syndrome during infancy in a patient with cystinosis. Acta Paediatr. 2006 Mar;95(3):379-80.

2006

Pediatr Res

Decreased intracellular ATP content and intact mitochondrial energy generating capacity in human cystinotic fibroblasts. Pediatr Res. 2006 Feb;59(2):287-92.

2006

Pediatr Nephrol.

Strict cysteamine dose regimen is required to prevent nocturnal cystine accumulation in cystinosis. Pediatr Nephrol. 2006 Jan;21(1):110-3. Epub 2005 Oct 27.

2005

Nephrol Dial Transplant

Altered status of glutathione and its metabolites in cystinotic cells. Nephrol Dial Transplant. 2005 Sep;20(9):1828-32. Epub 2005 Jun 14.

2004

Clin Chem

Comparison of cystine determination in mixed leukocytes vs polymorphonuclear leukocytes for diagnosis of cystinosis and monitoring of cysteamine therapy. Clin Chem. 2004 Sep;50(9):1686-8.

2002

Nephrol Dial Transplant

Follow-up and treatment of adults with cystinosis in the Netherlands. Nephrol Dial Transplant. 2002 Oct;17(10):1766-70.

Publications: Publications
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