Recently, we talked with Gary Frohlich, Senior Patient...
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The Rare Genomics Institute is a non-profit organization...
The Huntington's Disease Society of America (HDSA) holds...
Journal Watch: Soliris For Treating Neuromyelitis Optica
Possible good news for patients with neuromyelitis optica (aka Devic’s...
Jazz Pharmaceuticals Is Having a Good Year (Thanks to Its Strong Orphan Drugs Sales)
Chief financial officer and executive vice president of Jazz Pharmaceuticals...
Biomarin Update – Many Orphan Drugs On Target for Approval or Phase 3 Study
Chief financial officer of Biomarin, Dan Spiegelman, recently spoke at the...
Update on Insmed And Their Focus On Getting Arikace Approved For Two Indications
The treatment options for infections in patients with cystic fibrosis (CF)...
RDLA Legislative Round Up, May 2013
On Tuesday, Rare Disease Legislative Advocates (RDLA) hosted their May...
Jun 20 2013 to Jun 23 2013
Washington, D.C.
Jun 21 2013 to Jun 23 2013
Jacksonville, FL
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Many of Gaucher disease's presenting symptoms (hepatosplenomegaly, bone pain,...
The Huntington's Disease Society of America (HDSA) holds its annual convention...
In this exclusive interview with Rare Disease Report, the CEO of PSR Orphan...