Patients & Caregivers
Praveen Sethupathy Explores the Fibrolamellar Space
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.
FDA Hands Amphivena Orphan Drug Designation for New Approach to AML
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.
The Sickle Cell Association of New Jersey Talks Delayed Diagnosis and the Importance of Awareness
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.
Mogamulizumab's BLA Accepted for Filing and Priority Review Designation
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.
FDA Grants Breakthrough Therapy Designation to PellePharm for Gorlin Syndrome Treatment
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 Talks Neratinib in Fibrolamellar
Richard Bryce of Puma Biotechnology discusses his company’s interest in neratinib and working with fibrolamellar cancer.
Approval of Stutent Expanded to Reduce Carcinoma Recurrence
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.
FDA Gives Go-Ahead to Obinutuzumab for Follicular Lymphoma Treatment
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.
FDA Approves Hemlibra to Treat Bleeding in Hemophilia A Patients
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 Work in Fibrolamellar Carcinoma
John Gordan of UCSF discusses his experience in fibrolamellar, and what he’s been seeing with his most recent research.
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