In the months of May and June 2022, the European Medicines Agency (EMA) recommended granting various forms of marketing authorisation in the European Union (EU) for the following rare disease-targeted therapies: First therapy to treat two types of Niemann-Pick disease, a rare genetic metabolic disorder: Xenpozyme (olipudase alfa), a therapy for the treatment of non-central…
The Professional Society for Health Economics and Outcomes Research (ISPOR) is organising a webinar entitled “Health Preference Research in the Context of Gene Therapy in Rare Diseases” that will be limited to 1000 registered participants on a first-come, first-served basis. The webinar will take place on September 27, 2021 from 16.00 – 17.00 CET. This webinar will present the value…
Innovative techniques in the biomedical field (such as massive sequencing capacity, gene editing and tissue engineering) have resulted in the development of new unconventional drugs in the Advanced Therapy Medicinal Products (ATMPs) class. In this scenario, the European Paediatric Translational Research Infrastructure (EPTRI) and the EU-funded RESTORE project are collaborating to describe the map of competences available in the framework of the paediatric ATMP research in pan-European…
Based on evidence from a single-arm clinical trial, the European Medicines Agency (EMA) has recommended granting a marketing authorisation in the European Union for the gene therapy SKYSONA™ for the treatment of children with cerebral adrenoleukodystrophy (CALD), a severe form of a rare inherited neurological disease. SKYSONA™, developed by bluebird bio, is a one-time treatment which can…
EuropaBio invites you for its first webinar in the Advanced Therapies series of events, interviews, and publications titled « Taking health up a gear: Value of advanced therapies. » The webinar will take place on May 11, 2021 from 12.00 – 13.00 CET. The event will discuss how innovation in gene and cell-based medicine can bring a transformative value for patients, with the…