Nusinersen was approved as the first medical treatment for all patients with spinal muscular atrophy (SMA). A recent study in a large cohort of adult treated nusinersen demonstrated significant improvements Hammersmith functional motor scale expanded (HFMSE). However, clinically meaningful were seen only 40% at 14 months despite positive patients’ experience. The main issue open label remains r...

The term registry is widely used to refer to any database storing clinical information collected as a byproduct of patient care. Despite the use of this single characterizing term (registry), these databases exist in various forms and support functions ranging from biomedical informatics and clinical research, to public health, epidemiology and evidence-based clinical practice. This ambiguous t...

● In 2005, AIFA (Italian Drug Agency) implemented drug registries to ensure appropriate use of the treatment and enabling conditional agreements (i.e., risk-sharing) until the risk/benefit ratio of the drug is confirmed in clinical practice. ● Strong collaboration between AIFA, Clinicians, Hospital Pharmacists, Pharmacologists, Payers (Regional and Local) and pharmaceutical companies is needed ...

The four primary objectives of this descriptive study were to: 1) design a quality-measurement instrument for institutional cancer registries (ICRs), 2) evaluate the existing ICRs in Colombia with the designed instrument, 3) categorize the different registries according to their quality and prioritize efforts that will efficiently promote better registries with the limited resources available, ...

Clinical trial registries are being increasingly acknowledged worldwide. We searched for possibly trustworthy online registries that are not already included in the International Clinical Trials Registry Platform to evaluate whether other useful trial data sources exist and whether they could potentially be consulted, since the strategy search within this platform has recently been questioned. ...