Rare Disease Report
Patients & Caregivers


Praveen Sethupathy, PhD sat down with Rare Disease Report to discuss why it’s worth being excited about the new breakthroughs being made in fibrolamellar carcinoma.
Amphivena Therapeutics, Inc. was granted Orphan Drug Designation from the U.S. FDA for AMV564, its lead compound intended for the treatment of Acute Myeloid Leukemia.
Rare Disease Report sat down with Mary LaMar, founder and CEO of The Sickle Cell Association of America, and sickle cell disease patient Sakiyyah Darden who also works in an administrative support role for the organization.
The FDA has accepted for review the Biologics License Application for mogamulizumab to treat Cutaneous T-cell Lymphoma in patients who have received at least one prior systemic therapy.
The FDA granted Breakthrough Therapy Designation and Orphan Drug Designation to PellePharm for its compound topical patidegib for use in patients with Basal-cell nevus syndrome (BCNNS).
Richard Bryce of Puma Biotechnology discusses his company’s interest in neratinib and working with fibrolamellar cancer.
Stutent (sunitinib malate), which was developed by Pfizer and originally approved by the FDA in 2006 for gastrointestinal stromal tumors, received an approval expansion today.
On the same day that Genentech received U.S. FDA approval for emicizumab, the regulatory agency has also given the green light to Gazyva to treat follicular lymphoma.
The U.S. Food and Drug Administration (FDA) approved Genentech’s Hemlibra for patients with hemophilia A who have developed Factor VIII inhibiting antibodies.
John Gordan of UCSF discusses his experience in fibrolamellar, and what he’s been seeing with his most recent research.
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