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Patients Involved – An ultra orphan disease

Introduction A collaboration of Genzyme, International Pompe Association (IPA); UK, Dutch, and US Pompe patient organisations, Erasmus Medical Center and Duke University to establish appropriate measures to ensure the...

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Vaccines

Vaccines are biological preparations developed from modified versions of the micro-organisms that cause a disease. Vaccines improve or convey immunity to particular diseases, usually in an effort to prevent...

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Discovery and development of medicines

Once a target receptor molecule or an enzyme has been identified, scientists begin to look for potential compounds that will interact with the target to correct disease-related activity.These molecules...

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Medicines regulation in Europe

Medicines regulation is the basis for ensuring that only medicines of a high quality are marketed. In Europe, the European Commission proposes high-level legislation in the form of regulations...

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Interview with François Houÿez

Transcription I’m François. I’m 51. I work for the European organisation for rare disease EURORDIS, where my main role is to help patients navigating through the different procedures for...

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Patients Involved – Phase III Hepatitis C

Introduction This is a collaboration between Bristol-Myers Squibb (BMS) with FUNDHEPA (patient organisation) to give more patients the opportunity to receive an innovative treatment for Hepatitis C through the...

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