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A total of 342 posts are filed under Innovation
Rare Disease Report: FDA Fully Approves Iclusig for Chronic Myeloid Leukemia
(Source) The FDA has fully approved ARIAD Pharmaceuticals’ Iclusig (ponatinib) for the treatment of adult patients with chronic phase,… Continue Reading
Kindergarten Student with EB Organizes Awareness for Her Class
By Mark Guydish  Assume the largest organ in your body was on the fritz from birth. What do you think would be the most obvious problem?… Continue Reading
Genentech’s New Giant Cell Arteritis Drug Shows Majority of Users Achieved Steroid-Free Disease Remission at One Year
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced positive results from the Phase III GiACTA study, which… Continue Reading
N. Scott Adzick, MD, Children's Hospital of Philadelphia's Surgeon-in-Chief, Awarded the Congenital Hyperinsulinism International (CHI) "Be My Sugar" Award for Surgical Excellence (PRNewsFoto/The Children's Hospital of Phil)
N. Scott Adzick, MD, CHOP’s Surgeon-in-Chief, Recognized by Congenital Hyperinsulinism International (CHI)
As surgeon-in-chief at Children’s Hospital of Philadelphia (CHOP), and founder and director of the hospital’s Center for Fetal… Continue Reading
Repurposing Drugs for Rare Diseases: Collaboration is Key
by Tim Hoctor, Vice President, Corporate R&D Professional Services, Elsevier (SOURCE) The statistics are sobering: an estimated 350… Continue Reading
New Study Being Run for Catalyst Pharma for Patients with Congenital Myasthenic Syndromes (CMS)
Patients may now be eligible to participate in a new research study evaluating an investigational medicine for the treatment of CMS. WHY a… Continue Reading
Will Aetna Stop Providing an Approved Orphan Drug to Treat Rare Disease Patient?
by James Radke, PhD, thanks to Rare Disease Report for this piece. Orphan drugs are expensive. That is well established. However, the… Continue Reading