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...


Rare disease spotlight


If you were unable to join us in June for our first educational webinar geared to the #Dravet community - "Coping with Stress during Covid-19" - it is available on-demand!

Join us tomorrow, 7/30, at 3pm ET for updates on the upcoming EPX-100 (clemizole hydrochloride) Phase 1 & 2 Studies for #Dravet syndrome. Register to join live and ask questions or visit our website for the archived video after the event.

Attention Advocates! We need your help!

On July 23rd, #RDLA in coordination with the Rare Disease Congressional Caucus, will host a virtual briefing on "Rare Disease Community Experiences with #COVID19 Response Efforts and Future Policy Opportunities”.

That's a wrap!

Today, #RareAcrossAmerica advocates geared up for their meetings with Members of Congress by practicing their pitches! @SmithSolve #FastForwardforRare

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