The clinical trial is designed to enroll 6 patients with cystinosis currently being treated with cysteamine
to examine clinical parameters such as kidney function, muscle strength, bone density, and endocrine function.
- The US Food and Drug Administration has accepted Irish drug developer Horizon Therapeutics plc's (NASDAQ: HZNP) New Drug Application for Procysbi (Cysteamine
Bitartrate) Delayed-Release Oral Granules in Packets, the company said.
As a rare autosomal-recessive metabolic disorder, cystinosis is caused by defective transport of cystine across the lysosomal membrane. Once the diagnosis of cystinosis is confirmed, specific treatment with cysteamine
, an aminothiol, should be applied to the patient as soon as possible in order to preserve renal function and improve growth in affected children. Cysteamine
is the only specific drug approved by the Food and Drug Administration for cystinosis.
Under physiologic and Hixson-Crowell standard conditions, haloperidol (anti-psychotic) grafted CQDs with cysteamine
hydrochloride can be used for controlled release for up to 40 h.
and Sodium dodecyl sulfate (SDS) were obtained from Beijing Biotopped Science & Technology Co.
Autophagy inducer, cysteamine
(250 [micro]M; Sigma) was used as a positive control in the bactericidal and mucolytic experiments.
Malhotra, "Antibody immobilized cysteamine
functionalized-gold nanoparticles for aflatoxin detection," Thin Solid Films, vol.
Moreover, pharmacological inhibition of TG2 using cysteamine
attenuated anxiety- and depression-like behavioral abnormalities induced by chronic corticosterone treatment in mice .
The oocyte culture medium (OCM) consisted of bicarbonate buffered TCM 199 with 2 mM L-glutamine supplemented with 0.02 mg/ml cysteamine
, 1 IU/ml hCG, 0.1 IU/ml FSH, 100 ml/ml penicillin, 100 mg/ml streptomycin, 10% FBS (Fetal bovine serum, Gibco 10270), and 0.2 mM Na-Pyruvate.
An investigational medicine called RP 103, a form of cysteamine
, is in clinical trials.
The authors point out that asymptomatic cystinosis can be diagnosed by anterior segment examination, especially during routine eye examination, and emphasize the important responsibility ophthalmologists have in preventing future kidney failure by enabling early treatment with cysteamine
(see pages 292-295).