Tag: Rare Disease

A total of 363 posts are filed under Rare Disease
A Task for Tyler- Finding Treatments for SSADH and Hypotonia
Author: Jason Martin Life is always a task! But that task is amplified tenfold for someone like my son Tyler who suffers from an extremely… Continue Reading
2q23.1 Diagnosis Leads Mom to Start Online Boutique for Daughter’s Future
Author: Sarah Fister-Brewer In 2004, I embarked on my motherhood journey with a baby girl named Madeline. As most pregnant ladies, before… Continue Reading
Ionis and Akcea Form hATTR Partnership Worth up to $1.7 Billion
Rare Daily Staff Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, entered into an exclusive, worldwide agreement to license… Continue Reading
Flagship Pioneering Backs Foghorn Therapeutics with $50 Million to Develop New Class of Drugs to Regulate Genes
Rare Daily Staff Flagship Pioneering today said it made a $50 million commitment to Foghorn Therapeutics to discover and develop drugs based… Continue Reading
Sanofi Passes on Alnylam Primary Hyperoxaluria Type 1 Candidate
Rare Daily Staff Alnylam Pharmaceuticals said that Sanofi Genzyme has opted out of its rights to develop and commercialize Lumasiran,… Continue Reading
Sarepta Therapeutics Plans to Submit DMD Drug Golodirsen for FDA Approval by Year End
Rare Daily Staff Sarepta Therapeutics said that following its meeting with The U.S. Food and Drug Administration about the development… Continue Reading
Don’t Forget About the Adults
I am laying down thinking to myself why? So many whys to ask about. Why do I have a rare disease that nobody can treat? Why am I in so much… Continue Reading