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 with decision-makers, payers and other stakeholders, can national patient groups effectively and efficiently represent their patient community in a consistent strategic approach. To access the PLUS position statement click here.

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 aim is to be the voice of all people living with a rare disease and to give visibility to rare diseases in the global health agenda.
Johan Prevot, IPOPI’s Executive Director is also taking part in the launch meeting and will present IPOPI’s advocacy work at the international level.
The preliminary phase of RDI has been steered by EURORDIS and national rare disease alliances from the US (NORD), Canada (CORD), Japan (JPA), China (CORD), India (I-ORD), the Ibero-American pan-regional alliance (ALIBER) and the International Federation for Epidermolysis Bullosa (DEBRA International).

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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 more information see the official press release here.