John Crowley, CEO, Amicus Therapeutics is set to deliver a unique presentation at this years World #OrphanDrug Congress in Geneva. John’s approach to #clinicaltrialdesign during …
World #OrphanDrug Congress 2011 – Preliminary agenda available
Following the successful launch of the inaugural event, the World #OrphanDrug Congress 2011 is set to become one of the leading forums for the …
Repligen awarded incentives by US and Europe to develop #OrphanDrug for Spinal Muscular Atrophy
Repligen announced yesterday that it has been granted Fast Track and orphan drug status by the US FDA and the European Medicines Agency respectively for …
CSL Behring receives EU #OrphanDrug designations
CSL Behring has been granted Orphan Drug Designations (ODD) by the European Commission. Read the full article from Science 2.0 here. Orphan drug status was …
New discoveries shape future for #OrphanDrugs
The subject of orphan diseases is at times a very pessimistic one. These diseases are ones that affect fewer than 200,000 citizens, and although they …
Morphotek cancer drug granted #FDA orphan drug designation
Morphotek has been granted orphan drug designation by the US Food and Drug Administration (FDA) for their cancer drugs, MORAb-004 (for treatment of sarcoma) and …
Download presentation: The average cost of treatment for the #RareDisease AKU
The rare disease AKU or Alkaptonuria affects less than one in 250,000 people, but even so care for AKU patients should remain a priority in …
Makena backlash & potential impact on the #RareDisease community
Siren Interactive kindly provided this guest blog post from their blog, SirenSong. The publicity surrounding KV Pharmaceutical’s pricing and handling of the drug, Makena, is …