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Pharmacological treatment of nephropathic cystinosis with cysteamine


R Kleta, WA Gahl - eop, 2004 - informahealthcare.com
Cystinosis, clinically recognised since 1903, is an autosomal recessive lyso-
somal storage disease caused by mutations in CTNS. This gene codes for a
lysosomal cystine transporter, whose absence leads to intracellular cystine ...
Cited by 23 - Related articles - All 5 versions

Probes of the catalytic site of cysteine dioxygenase

- jbc.org
SC Chai, JR Bruyere, MJ Maroney - Journal of Biological Chemistry, 2006 - ASBMB
The first major step of cysteine catabolism, the oxidation of cysteine to
cysteine sulfinic acid, is catalyzed by cysteine dioxygenase (CDO). In the
present work, we utilize recombinant rat liver CDO and cysteine derivatives ...
Cited by 12 - Related articles - BL Direct - All 8 versions

Ophthalmic manifestations and histopathology of infantile nephropathic cystinosis: report of a …

- nih.gov
E Tsilou, M Zhou, W Gahl, PC Sieving, CC … - Survey of ophthalmology, 2007 - Elsevier
Cystinosis is a rare autosomal recessive metabolic disorder characterized by the
intracellular accumulation of cystine, the disulfide of the amino acid cysteine,
in many organs and tissues. Infantile nephropathic cystinosis is the most ...
Cited by 4 - Related articles - All 13 versions

Hematological Manifestations of Nephropathic Cystinosis


A Emadi, KH Burns, B Confer, MJ Borowitz, MB … - Acta Haematol, 2008 - content.karger.com
Pancytopenia is an uncommon manifestation of cystinosis, a congenital lysosomal
storage disease. We describe a 34-year-old patient with nephropathic cystinosis
with multisystem involvement who developed progressive bone marrow failure ...
Related articles - All 5 versions

Formulation and in vitro evaluation of cysteamine hydrochloride viscous solutions for the …


S Bozdağ, K Gümüş, Ö Gümüş, N Ünlü - European Journal of Pharmaceutics and …, 2008 - Elsevier
In the present study, viscous solutions of cysteamine hydrochloride (CH) were
prepared by using 0.5%, 1.0%, 1.5% or 3.0% of hydroxypropylmethylcellulose
(HPMC) and were evaluated for their in-vitro characteristics and stability. ...
Related articles - All 2 versions

[PDF] Ocular Manifestations in Adolescent Cystinosis: Case Report in Thailand


N Kitnarong, P Osuwannaratana, W … - J Med Assoc Thai, 2005 - medassocthai.org
Correspondence to : Kitnarong N, Department of Ophthal- mology, Faculty of
Medicine, Siriraj Hospital, Mahidol University, 2 Prannok, Bangkok Noi, Bangkok
11000, Thailand. Phone: 0-2419-8033, Fax: 0-2411-1906, E-mail: ...
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Evaluation of Treatment With Cysteamine Eyedrops for Cystinosis With Confocal Microscopy


R Tavares, D Coelho, MC Macário, A Torres, … - Cornea, 2009 - journals.lww.com
Methods: A 20-year-old woman with infantile cystinosis, with a history of kidney
transplantation at age 10, was studied. She applied cysteamine eyedrops
(0.1136%) 10 times a day (a new formulation, now stable at room temperature ...
Related articles - All 3 versions

41 Cystinosis


WA Gahl - Springer
Nephropathic cystinosis (1–3) deserves a special place in the annals of
clinical medicine as the first treatable lysosom- al storage disease. The
pathophysiology itself, based upon the formation of cystine crystals within ...
Related articles


 


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