Access to the themed issue from Expert Opinion on Orphan Drugs: Rare disease - Detection, Treatment, Care, and Reasearch, Informa Healthcare

Access to the themed issue from Expert Opinion on Orphan Drugs: Rare disease - Detection, Treatment, Care, and Reasearch, Informa Healthcare

OTRAS NOTICIAS DE LAS ENFERMEDADES RARAS

Descubre lo nuevo en el área de las enfermedades raras Hasta el 16 de marzo – acceso LIBRE a la edición del diario Expert Opinion sobre el tema de las enfermedades raras To raise awareness for Rare Disease Day, Expert Opinion on Orphan Drugs are offering FREE access to our recent themed issue titled Rare diseases - Detection, Treatment, Care and Research until March 16th, 2015. Click on the links below to access the articles. INTERVIEW Working collaboratively and internationally to improve the lives of people affected by rare disease EDITORIAL A hidden priority: the paradox of rarity (EURORDIS perspective) A hidden priority: the paradox of rarity (NORD perspective) The diagnostic gap – an expert opinion Access and availability of orphan drugs in the United States: advances or cruel hoaxes? A US perspective on newborn screening: a powerful tool for prevention Working with the NHS to develop the Alström multi-disciplinary clinic service Genethon: patient-empowered research Partnering in medical education: rare disease organizations bring experts and a patient voice to the conversation Findacure: empowering patient groups for drug development The orphan framework as a new opportunity: an expert opinion The Progeria Research Foundation: its remarkable journey from obscurity to treatment How close are we to a cure for Duchenne muscular dystrophy? Resources PATIENT STORY Adventures of an advocate: Annette De Bow’s big journeys (Patient Story) Knowing more about Noonan, by Samantha Scheer (Patient Story) Stiff person syndrome, by Liz Blows (Patient Story) REVIEW  Pulmonary arterial hypertension: a rare disease that encourages the development of multiple treatments The emergence of gene therapy for rare diseases The impact of integrated omics technologies for patients with rare diseases Resources This themed issue has been organised in partnership with EURORDIS (European Organisation for Rare Diseases) and NORD (National Organization for Rare Disorders). The purpose of this themed issue is to improve the scientific community’s understanding of the important issues surrounding rare diseases and the impact they have on the lives of patients and their loved ones. Throughout, it highlights areas where it makes sense to work collaboratively and at the international level. If you have any questions about any of these papers or queries about the journal, please contact Lizzie Howard.  PLEASE NOTE, ALL ARTICLES MENTIONED ABOVE ARE FREE UNTIL MARCH 16TH 2015 Read More: http://informahealthcare.com/page/free_access_EOOD