Entries by IPOPI
The Value of One Patient Organisation per Country
PLUS, the Platform of Plasma Protein Users, which represents organisations of patients living with rare plasma related disorders and rare bleeding disorders, recently issued a position statement on the value of one national patient organisation per country. The statement highlights that only by having a single recognised patient organisation that channels the views of patients […]
Rare Diseases International launched today
Over 60 patient representatives from 30 countries are gathering this afternoon in Madrid, Spain, for the inauguration of Rare Diseases International (RDI) and to adopt a joint declaration to advocate for rare diseases as an international public health priority. This Eurordis initiative represents patients and families of all nationalities across all rare diseases and its […]
Marketing application for new ADA-SCID gene therapy submitted to EMA
GSK (GlaxoSmithKline), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) recently announced the submission of a marketing application to the European Medicines Agency (EMA) for a gene therapy (GSK2696273) to treat patients with adenosine deaminase severe combined immunodeficiency syndrome (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. For […]
EU-wide overview of the market of blood, blood components and plasma derivatives
The European Commission (EC) published in April a report on ‘An EU-wide overview of the market of blood, blood components and plasma derivatives focusing on their availability for patients’. The key objective of the document is to provide an economic perspective on the European blood sector. This was done against the background of understanding and […]
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