Latest News

  • Alnylam and Sanofi Enter into Strategic Restructuring of RNAi Therapeutics Rare Disease Alliance
    Tuesday, January 9, 2018
    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...
  • Zymeworks Highlights Key 2017 Accomplishments and Announces 2018 Priorities
    Tuesday, January 9, 2018
    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,...
  • We Might Finally Know How Alzheimer’s Spreads Through The Brain
    Tuesday, January 9, 2018
    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...
More News

Videos

2147477740
Rare Conversations: Sheri & Stephanie
2147477595
Rare Conversations: Kendra & Preston
2147477524
Overview on Understanding Mitochondria?
More Videos

Rare disease spotlight

Neonatal alloimmune thrombocytopenia
Ubiquitin Disorder
Pseudo-Zellweger syndrome
Pterygium colli mental retardation digital anomalies
More Rare Diseases

Twitter

Rare DiseasesFollow

CheckOrphan is the leading #news and information platform dedicated to #rarediseases and #orphandrugs

Rare Diseases
Retweet on TwitterRare Diseases Retweeted
curedravetDravet Syndrome Foundation@curedravet·
29 Jul

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. https://bit.ly/3hJOXY0

Reply on Twitter 1288473397228441601Retweet on Twitter 12884733972284416015Like on Twitter 128847339722844160111Twitter 1288473397228441601
Retweet on TwitterRare Diseases Retweeted
RareAdvocatesRDLA@RareAdvocates·
21 Jul

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”.

Reply on Twitter 1285588730800218112Retweet on Twitter 12855887308002181121Like on Twitter 12855887308002181121Twitter 1285588730800218112
Retweet on TwitterRare Diseases Retweeted
RareAdvocatesRDLA@RareAdvocates·
21 Jul

That's a wrap!

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

3
Reply on Twitter 1285692020435148805Retweet on Twitter 12856920204351488052Like on Twitter 12856920204351488056Twitter 1285692020435148805
Retweet on TwitterRare Diseases Retweeted
sicklecell101Sickle Cell 101@sicklecell101·
15 Jul

"I've seen parents blame their children for having #sicklecell disorder & falling ill all the time. As a healthcare provider and patient, I have to speak up. I am motivated to educate people to reduce stigmatization." Read more: http://sc101.org/scay20 #SCAYAwards #sicklecell101

Reply on Twitter 1283476852951207937Retweet on Twitter 12834768529512079373Like on Twitter 128347685295120793712Twitter 1283476852951207937
Load More...