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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...

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To save the 352,400 patients diagnosed each year in the USA with rare or orphan cancers we must save small biotech firms who provide these drugs. The SEC needs to GET SHORTY! @RareDiseases #mmsm @CheckOrphan @rarediseasefdn @SEC_Enforcement @SteveFDA

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ICYMI: During XLH Week we talked about the Transitions Toolkit, a collaboration between Quinnipiac University, Ultragenyx and The XLH Network, Inc.. The toolkit is available for download at our website: https://xlhnetwork.org/xlh-resources

We’re honored to receive the @Rarediseases 2020 Industry Innovation Award at this year’s #RareImpact Awards. Our commitment to reimagining medicine has led to innovative treatments for children like Malachi, who has #SpinalMuscularAtrophy (SMA) Type 1.

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