'Rare Disease-Detection, Treatment, Care and Research'

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‘Expert Opinion on Orphan Drugs:

Rare Disease-Detection,

Treatment, Care and Research’

As a EURORDIS Member, the MEBO community can access a special edition of the journal 'Expert Opinion on Orphan Drugs: Rare Disease-Detection, Treatment, Care and Research' for free until 26 December. As the EURORDIS Junior Communications Manager, tells MEBO in an email,

Please use the following link to access the journal online:

http://informahealthcare.com/page/free_access_EODD

This themed issue has been organised in partnership with us, EURORDIS, and NORD. 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. Our CEO, Yann Le Cam has contributed an article entitled ‘A hidden priority: the paradox of rarity (EURORDIS perspective)’ to the edition, available at the above link.

The issue includes pieces covering the importance of raising awareness and sharing family experiences on coping and caring for a child affected by a complex rare disease; creating patient communities; promoting policies and support in favour of research on rare and genetic diseases; promoting better standards of diagnosis and care; and easier access to cross-border care for patients affected by rare diseases.

For a limited time, you can access content from this themed issue for Free below

• Working collaboratively and internationally to improve the lives of people affected by rare disease (Interview)
• A hidden priority: the paradox of rarity (EURORDIS perspective) (Editorial)
• A hidden priority: the paradox of rarity (NORD perspective) (Editorial)
• Adventures of an advocate: Annette De Bow’s big journeys (Patient Story)
• The diagnostic gap – an expert opinion (Editorial)
• Knowing more about Noonan, by Samantha Scheer (Patient Story)
• Pulmonary arterial hypertension: a rare disease that encourages the development of multiple treatments (Review)
• Access and availability of orphan drugs in the United States: advances or cruel hoaxes? (Editorial)
• A US perspective on newborn screening: a powerful tool for prevention (Editorial)
• Working with the NHS to develop the Alström multi-disciplinary clinic service (Editorial)
• Stiff person syndrome, by Liz Blows (Patient Story)
• Genethon: patient-empowered research (Editorial)
• Partnering in medical education: rare disease organizations bring experts and a patient voice to the conversation (Editorial)
• Findacure: empowering patient groups for drug development (Editorial)
• The orphan framework as a new opportunity: an expert opinion (Editorial)
• The Progeria Research Foundation: its remarkable journey from obscurity to treatment (Editorial)
• The emergence of gene therapy for rare diseases (Review)
• The impact of integrated omics technologies for patients with rare diseases (Review)
• How close are we to a cure for Duchenne muscular dystrophy? (Editorial)
• Resources

PLEASE NOTE THESE ARTICLES ARE FREE TO ACCESS UNTIL DECEMBER 26TH 2014

Read More:http://informahealthcare.com/page/free_access_EODD

We appreciate the invaluable work EURORDIS and NORD have done for all rare disease patients and organizations throughout the year. We are proud to be members of these organizations.

Maria


María de la Torre
Founder and Executive Director

A Public Charity
www.meboresearch.org
www.brasil.meboresearch.org
maria.delatorre@meboresearch.org
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