FDA Outlines a Collaborative Approach for Pediatric Rare Disease Trials

Earlier this month the U.S. Food and Drug…

Give HOPE for the Holidays
  We welcome the holiday season, a time for family and friends, joy and HOPE. As 2017 comes to a close, we celebrate RARE families and give… Continue Reading
FDA Lifts Clinical Hold on Alnylam’s and Sanofi’s Hemophilia Studies of Fitusiran
Rare Daily Staff Alnylam Pharmaceuticals and Sanofi Genzyme said the U.S. Food and Drug Administration has lifted the hold on clinical… Continue Reading
Amicus Therapeutics Seeks FDA Approval for Migalastat to Treat Fabry Disease
Rare Daily Staff Amicus Therapeutics said it submitted a new drug application to the U.S. Food and Drug Administration to seek approval for… Continue Reading
FDA Approves Expanded Use of GSK’s Nucala to Include EGPA
GlaxoSmithKline said that the U.S. Food and Drug Administration has approved Nucala as the first targeted treatment for eosinophilic… Continue Reading
Global Genes Rare Disease Stock Index Ends the Week up 0.33 Percent
Markets ended the week slightly higher on positive job news. Eyes are on Washington, D.C. as investor watch how the House and Senate will… Continue Reading
Lost in Translation
If you want to understand the value of patient-centered outcome measures, consider people with the rare progressive muscle disease Duchenne… Continue Reading
Alexion Licenses Halozyme Drug Delivery Technology in Deal Worth up to $680 Million
Rare Daily Staff Alexion Pharmaceuticals entered into a licensing agreement valued at up to $680 million with Halozyme Therapeutics to use… Continue Reading
Spark and Pfizer Gene Therapy Shines in NEJM Report of Interim Data from Study in Hemophilia B
Rare Daily Staff Interim data from a phase 1/2 study of Spark Therapeutics’ and Pfizer’s experimental gene therapy SPK-9001 for… Continue Reading
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