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  • 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...


Rare disease spotlight


NORD @RareDiseases has released the results of The #COVID19 Community Survey Report, showing impact on #raredisease patients and their families. To access this important report from NORD, click here:

Only 1 week left to nominate someone for the SCAY Awards and giveaway!

There are individuals within the #sicklecell community who work tirelessly for the cause, but their work goes unnoticed. We're celebrating them!

#sicklecell101 #scayawards


What do aHUS patients & their families really want?
H. O. P. E.
#RareDisease atypical HUS #aHUS #SHUa #KidneyDisease #genetic #CKD #atypicalHUS #patients #inspire #caregivers #advocacy #Motivation #PositiveThinking

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