Teenagers and young adults with nephropathic cystinosis display significant bone disease and cortical impairment.
Background: Bone impairment appears to be a novel complication of nephropathic cystinosis despite cysteamine therapy. Its exact underlying pathophysiology is nevertheless unclear.
Nephropathic cystinosis is a rare lysosomal storage disorder. Patients present in the first year of life with renal Fanconi syndrome that evolves to progressive chronic kidney disease (CKD).
In the cornea, crystalline, gold-dust deposition of cystine leads to visual impairment, recurrent erosions, photophobia, epiphora and blepharospasmus.
In this review, we aim to provide an overview of the latest advances in the pathogenetic, clinical, and therapeutic aspects of cystinosis.
Cystinosis is a rare autosomal recessive lysosomal storage disorder caused by mutations in the CTNS gene. Main dysfunction is a defective clearance of cystine from lysosomes that leads to accumulation of cystine crystals in every tissue of the body.
Effectiveness, safety and cost of abiraterone acetate in patients with metastatic castration-resistant prostate cancer: a real-world data analysis.
This study aims to evaluate the effectiveness, safety and cost of abiraterone acetate (AA) in patients with mCRPC.
Abiraterone is effective and should be considered for the treatment of metastatic castrate-naïve prostate cancer.
In this key paper evaluation, the authors discuss the rational, trial design and results of the LATITUDE trial. Furthermore, the past and current standard of care of metastatic castrate-naïve prostate cancer (mCNPC)...
Herein, the in-detail pharmacological profile of AA, including androgen signaling, mechanism of action of AA, mechanism of AA resistance, pharmacokinetics, latest clinical findings...
Potential nephrotoxicity of sofosbuvir-based treatment in patients infected with hepatitis C virus: a review on incidence, type and risk factors.
Introduction: There was no report of nephrotoxicity during clinical trials assessed sofosbuvir for treatment of hepatitis C (HCV) infection. This may be due to excluding patients with severe kidney dysfunction...
Aims: To evaluate the effect of dulaglutide on body composition in type 2 diabetes mellitus (T2DM) patients undergoing hemodialysis (HD).