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This article summarizes the milestones in the development of burosumab leading to its first global approval in the EU for XLH in paediatric patients.

The FDA on 1st May 2012 approved Elelyso (taliglucerase alfa) from Pfizer for long-term enzyme replacement therapy to treat a...

We sought to determine the efficacy of high-dose liraglutide (3.0 mg once daily) in patients with prior bariatric surgery.

Introduction: This phase III, double-blind, randomised, placebo-controlled trial (and extension phase) was designed to assess the efficacy and safety of velmanase alfa (VA) in alpha-mannosidosis (AM) patients.

Introduction: Long-term outcome data provide important insights into the clinical utility of enzyme replacement therapies.

Alpha-mannosidosis is an ultra-rare monogenic disorder resulting from a deficiency in the lysosomal enzyme alpha-mannosidase, with a prevalence estimated to be as low as 1:1,000,000 live births.

Sanofi and its subsidiary Genzyme announced that the FDA has granted a six-month Priority Review designation to its New Drug...

Sanofi and its subsidiary Genzyme announced that the European Commission (EC) has granted marketing authorization for Cerdelga (eliglustat) capsules, a...

Objectives: To establish a novel approach to classify fibrinogen disorders, we investigated the potential of clot waveform analysis (CWA) of CFA and searched for a surrogate marker for fibrinogen Ag.

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion...