I remain confident that these internal discussions will allow FDA to do the right thing, and grant eteplirsen approval.
Isabelle Jordon, Chair of the Rare Disease Foundation, talks about the origins of this unique and very effective organization.
The media attention received by the Zika outbreak is already raising people’s concerns about both the virus and a potential vaccine.
In the small number of years since the organization began, CDCN has succeeded in transforming the ways researchers fundamentally think about Castleman disease
FDA has had the NDA for eteplirsen since August 25, 2015. A normal review process for an orphan drug is 6 months.
While contact sports like boxing and American football are often associated with chronic traumatic encephalopathy (CTE), those are not the only sports that can lead to CTE.
Orphan drugs account for 5% of cost but rare diseases account for 9% of the population.
Gord Downie, lead singer of the Canadian rock band Tragically Hip has been diagnosed with glioblastoma.
OFF!, long sleeves, and condoms—those are the 3 main lines of defense that the US Olympic athletes will have against the Zika virus.
Vtesse announced that the dose-finding portion of the study is now complete.