IPF Patients Treated with Ofev® VS Placebo Were Twice as Likely to Have Improved or Stable Lung Function

Separate subgroup analysis demonstrates long-term…

Your Questions Answered on Glut1 Deficiency Syndrome
Talk to your doctor about Glut1 Deficiency Syndrome Many neurologic conditions share the symptoms of Glucose Transporter Type 1 deficiency… Continue Reading
As Her Boys Battle Rare Disease, Mom Drives for a Cure
There are five people in the United States that live with Jansen’s Metaphyseal Chondrodysplasia. Neena Nizar and her two sons, Arshaan and… Continue Reading
RareCAST: Experimental Rare Disease Therapy Wins New FDA Reg Med Designation
  Last month the U.S. Food and Drug Administration granted Enzyvant both Breakthrough Therapy Designation and the newly established… Continue Reading
Abeona Therapeutics Announces New Updates on Sanfilippo Syndrome Clinical Trial
Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for… Continue Reading
Catalyst Pharmaceuticals Receives 2017 Beacon Council Award for Contributions in Life Sciences & Healthcare
Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for… Continue Reading
Biotech Leader and Rare Disease Champion, Henri Termeer, Passes at Age 71
This last week, the Rare Disease Community lost a true Champion of Hope.  The announcement of Henri Termeer’s passing was met with shock… Continue Reading
FindaCure Workshop Agenda: A-Z To Setting Up a Patient Group’ Workshop
Findacure and Genetic Alliance UK are pleased to release the schedule for their upcoming workshop on setting up a patient group. The… Continue Reading

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