A randomised placebo-controlled trial of topical cysteamine therapy in patients with nephropathic cystinosis
نویسندگان
چکیده
منابع مشابه
Removal of corneal crystals by topical cysteamine in nephropathic cystinosis.
In patients with nephropathic cystinosis, corneal crystals develop by one year of age; they progressively accumulate and eventually cause recurrent corneal erosions and photophobia. After an in vitro study of cystinotic corneal stromal cells showed cystine depletion by cysteamine and after topical cysteamine was determined to be nontoxic in rabbits, we performed a controlled double-blind clinic...
متن کاملPopulation pharmacokinetics and pharmacodynamics of cysteamine in nephropathic cystinosis patients
BACKGROUND Nephropathic cystinosis is an autosomal recessive disorder resulting in an impaired transport of cystine trough the lysosomal membrane causing an accumulation of free cystine in lysosomes. The only specific treatment for nephropathic cystinosis is cysteamine bitartrate. This study was aimed to describe the relationship between cysteamine plasma concentrations and white blood cell cys...
متن کاملSwallowing dysfunction in 101 patients with nephropathic cystinosis: benefit of long-term cysteamine therapy.
Nephropathic cystinosis is a rare, autosomal recessive lysosomal storage disorder caused by mutations in the CTNS gene that codes for a cystine transporter in the lysosomal membrane. Affected patients store 50-100 times the normal amounts of cystine in their cells, and suffer renal tubular and glomerular disease, growth retardation, photophobia, and other systemic complications, including a myo...
متن کاملClearance of corneal crystals in nephropathic cystinosis by topical cysteamine 0.5%.
A 2-year-old girl with nephropathic cystinosis was successfully treated with topical cysteamine 0.5% to one eye. Clearance of crystals from the treated cornea was virtually complete after three months. The possibilities and limitations of this form of treatment are discussed.
متن کاملNephropathic cystinosis in adults: natural history and effects of oral cysteamine therapy.
BACKGROUND The full burden of nephropathic cystinosis in adulthood and the effects of long-term oral cysteamine therapy on its nonrenal complications have not been elucidated. OBJECTIVE To assess the severity of cystinosis in adults receiving and not receiving oral cysteamine therapy. DESIGN Case series. SETTING National Institutes of Health Clinical Center. PATIENTS 100 persons (58 men...
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ژورنال
عنوان ژورنال: Eye
سال: 1991
ISSN: 0950-222X,1476-5454
DOI: 10.1038/eye.1991.139