Rare Disease Report


The New England Journal of Medicine has published interim data from Spark Therapeutics' Phase 1/2 clinical trial of SPK-9001 in patients with hemophilia B.
In this video, Kevin Sullivan, M.D., discusses why he and his group have an interest in this space, as well as what challenges he’s had in working with a rare cancer type.
Richard Cutler of Puma Biotechnology is hopeful that neratinib, which was approved for breast cancer in July, can provide clinical benefit to rare liver cancers.
A Phase 3 of Imbruvica in combination with Rituxan in relapsed/refractory and treatment-naïve patients with Waldenstrom’s macroglobulinemia has successfully met its primary endpoint.
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.
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