uniQure N.V. announced that it will not pursue the renewal of the Glybera(alipogene tiparvovec) marketing authorization in Europe when it...
The Committee for Medicinal Products for Human Use (CHMP) has refused, a second time, to approve the gene therapy product...
This article summarizes the milestones in the development of burosumab leading to its first global approval in the EU for XLH in paediatric patients.
Cached in a SEC filing (9 April), but not referred to in press releases, is a summary of a EMA...
In this review, we aim to provide an overview of the latest advances in the pathogenetic, clinical, and therapeutic aspects of cystinosis.
uniQure announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion...
Objectives: To summarize available clinical evidence for cysteamine bitartrate preparations in the treatment of nephropathic cystinosis as identified through a systematic literature review (SLR).
Gaucher disease is an inherited metabolic disease characterized by β-glucocerebrosidase deficiency and commonly treated with enzyme replacement therapy (ERT). The efficacy of ERT with velaglucerase alfa was assessed based on...
Cystinosis is the most common hereditary cause of renal Fanconi syndrome in children.
Cystinosis is a rare autosomal-recessive lysosomal storage disease with high morbidity and mortality. It is caused by mutations in the CTNS gene that encodes the cystine transporter, cystinosin, which leads to lysosomal cystine accumulation.