Latest News
-
PARIS, France and CAMBRIDGE, MA, USA – Sanofi and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced a strategic restructuring of their RNAi therapeutics alliance to streamline and optimize development and commercialization of certain products for the treatment of rare genetic diseases. Specifically: Sanofi will obtain global development and commercialization rights... -
VANCOUVER, British Columbia – Zymeworks Inc. (NYSE/TSX: ZYME), a clinical-stage biopharmaceutical company developing next-generation multifunctional biotherapeutics, provided key accomplishments from the past year and announced priorities for 2018. “2017 was a great year for Zymeworks and we are poised to achieve a number of important corporate milestones in 2018,” said Ali Tehrani,...
-
Alzheimer’s disease is a devastating brain illness that affects an estimated 47 million people worldwide. It is the most common cause of dementia in the Western world. Despite this, there are currently no treatments that are effective in curing Alzheimer’s disease or preventing its relentless progression. Alzheimer’s disease is caused...
Videos
Rare disease spotlight
At @bandage_bcn we are team with researchers from @GeneticsUB and @SJDbarcelona_es and one of our projects is focused on MAGEL2 mutations.
@RareConnect @rarediseaseuk @eurordis @revistanewsrare @CheckOrphan @BLACKSWANFound @RareDiseases @react_community @fpwr @corrales_noelia https://twitter.com/bandage_bcn/status/1278611572466860032
#MAGEL2 syndrome (or SYS) is a severe #raredisease characterized by developmental delay, feeding difficulties, & hypotonia among others. Not a cure have been find yet and thus, research is the only hope for this patients for a better life.
@CIBERER
https://scienceandspice.wordpress.com/2020/07/02/magel2-and-schaaf-yang-syndrome/
Late this afternoon, the FDA approved Fintepla for use in Dravet syndrome! Congratulations to @zogenix and thank you to all of our families who participated in the trials and helped bring this to fruition.
https://www.prnewswire.com/news-releases/fda-approves-new-therapy-for-dravet-syndrome-301084154.html
