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European Patient Advocacy Group patient representatives have been announced

EURORDIS created the ePAGs to engage patient organisations and ensure a democratic process of patient representation in the decision-making processes around European Reference Networks (ERNs).

May 17, 2016

EURORDIS has developed ePAGs for each ERN disease grouping. ePAGs bring together elected patient representatives and affiliated organisations who will ensure that the patient voice is heard throughout the ERN decision-making processes and a democratic process of patient representation.

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The FDA designates “lentiviral vector containing human liver and erythroid pyruvate kinase (PKLR) gene” as new Orphan Drug for the PKD treatment

The orphan drugs designation by the FDA, together with the one achieved by the European Medicines Agency in 2014, will allow promoters to get benefits for the development of this lentiviral vector to facilitate the approval for its commercialization

May 9, 2016

The Food and Drug Administration (FDA), the govern agency of United States responsible for the drugs regulations, has designated “lentiviral vector containing human liver and erythroid pyruvate kinase (PKLR) gene” as new Orphan Drug for the PKD treatment, a rare anaemia caused by mutations in the gene that codifies the pyruvate kinase enzyme, which impairs directly the metabolic energy of red cells and results in the reduced lifespan and early breakdown of erythrocytes in the blood.

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ThalCare tool for the management of patients with thalassaemia

The tool assists medical and nursing teams enabling the centers to collaborate with multidisciplinary experts on-line

April 20, 2016

ThalCare provides information and treatment assistance by tracking transfusions, chelation, vaccination and other key related issues. Moreover, ThalCare enables prevention of the disorder by cascade screening methodology and assesses the risk category for transplants indicating suitability for BMT.

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ENERCA e-Learning platform is already available!

To access to the e-Learning platform you only have to freely register, learn with us!

April 8, 2016

The ENERCA e-Learning platform is a useful tool for initial and continuing medical education in the field of rare anaemias, offering a complete panel of training tools to the registered participants and contributing to lowering inequalities in knowledge accessibility throughout Europe. To participate you only have to freely register. Join, enjoy and learn with us!  

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Register for Rare Barometer Voices: Make your voice heard!

Rare Barometer Voices is part of the new EURORDIS initiative, the Rare Barometer Programme.

March 22, 2016

EURORDIS, the European Organisation for Rare Diseases, has developed the Rare Barometer Voices, a new interactive survey panel available in 23 languages that collects the experiences of people that are living with or affected by a rare disease.

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Join the ''TEST2CONTROL'' WEBINAR organized by TIF & Bio Rad Laboratories

8 May 2019 INTERNATIONAL THALASSAEMIA DAY

8 May 2019

Although thalassemia and sickle cell are among the most common monogenic haemoglobin disorders in the world, disease awareness is still far from optimal. In commemoration of International Thalassaemia Day 2019 on May 8th, TIF & Bio Rad Laboratories Inc. invite you to register for our #Test2Control Thalassemia and Sickle Cell Webinar about the past, present, and future of monitoring and treating thalassaemia and other haemoglobinopathies.  Dr. Michael Angastiniotis, Medical Advisor, Thalassaemia International Federation (TIF) & Dr. Daniel E. Bauer, MD PhD

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Call for interest for European Reference Networks will be launched 16th March!

The date of the launch of the call for interest for ERNs has been confirmed as 16 March 2016 and will be divided into two waves

March 14, 2016

Moreover, 7th April an Info Day will be held in Brussels to explain the call for ERN to the potential interested network applicants and to answer questions on this call.

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2016 EURORDIS Awards for Excellence in the Field of Rare Diseases

EURORDIS presented last 23th February its 2016 Awards for Excellence in the Field of Rare Diseases at Black Pearl Gala Dinner

February 26, 2016

The ceremony took place in Brussels last 23rd February within the Black Pearl evening, presented by Royal Highness Princess Astrid of Belgium and in presence of Rare Disease Day Ambassador Sean Hepburn Ferrer. The Awards recognizes outstanding patient advocates and organisations, volunteers, scientists, companies, media and policy makers who have contributed to reduce the impact of rare diseases on people's lives through the EURORDIS Awards for Excellence in the field of Rare Diseases.

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2016 Rare Disease Day video out now! Do not forget: 29th February... Rare Disease Day!

With the theme “Patient Voice” and the slogan “Join us in making the voice of rare diseases heard” hundreds of events will be held around the world

February 2, 2016

In its ninth edition, the Rare Disease Day will recognize the essential role that patients play in voicing their needs and inviting to join rare disease community to all those that are not directly affected by a rare disease avoiding the isolation of patients and families.

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RD-ACTION launches an informal ERN ‘Matchmaker’ tool

The tool aims to facilitate discussions and collaboration between experts willing to join an ERN.

January 21, 2016

The new Joint Action for Rare Diseases has launched an informal Rare Disease ERN “matchmaker” exercise to facilitate discussions and collaboration between experts willing to join an ERN. At intervals of approximately 3 weeks a spreadsheet of the responses received for each Thematic Grouping will be emailed to all those submitting their data under that same Thematic Group.

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