Tags » Rare Disease

RARE DISEASE: Awareness, favoured children and the Layered for Cryo Challenge

The Rare Disease Foundation defines a rare disease as “a condition affecting fewer than 1 in 2000 people”. They go on to estimate that about 1 in 12 Canadians are diagnosed with one of 7000+ different rare diseases. 566 more words


There is no Instant Gratification in Rare Disease

I am an instant gratification kind of gal.  Who isn’t?  We are all used to having everything at our fingertips all of the time.  So, I didn’t expect anything different with Bug’s newest round of testing.  770 more words

Rare Disease Day 2015

February 28, 2015, will be the 8th Rare Disease Day. The event is created and coordinated by EURORDIS along with many international rare disease National Alliances. 144 more words

Rare Diseases

Orphan Drugs: Free Mobile App Launched By Coté Orphan

Coté Orphan, LLC, a regulatory affairs advisory firm located in Silver Spring, Maryland, announces the launch of a FREE orphan drug mobile app, OrphanDB, that provides the user with up-to-date information on orphan drug designation status for two regulatory agencies: 138 more words

Orphan Drug Development

Vertex Pharmaceuticals: FDA Advisory Committee Recommends Kalydeco For R117H CF Mutation

On October 21st, the FDA’s Pulmonary Allergy Drugs Advisory Committee (PADAC) recommends by a vote of 13 to 2, the approval of orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis (CF) patients ages 6 and older who have the R117H mutation in the CF Transmembrane Regulatory (CFTR) gene. 250 more words

Orphan Drug Development

FDA Orphan Drug Designations: 2014 9-Month Approval Rate

The chart below compares the number of FDA Orphan Drug Marketing Approvals in the first 9 months of 2013 versus the number of FDA ODD Marketing Approvals in the first 9 months of 2014. 425 more words

Orphan Drug Development

A Perfect Monster

“Nothing is so painful to the human mind as a great and sudden change.”
Mary Shelley, Frankenstein

Alright, alright, I get it life, unexpectedly parenting a sick child knocks the wind, startles the soul, evolves the identity but you know what?

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