Rare Disease Report


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.
New Phase 1 clinical data for BLU-285 supports previous data that demonstrated the drug as exhibiting strong clinical activity and an encouraging safety profile.
Bristol-Myers Squibb announced that the FDA has expanded its approval of Sprycel tablets. The new indication includes children with rare chronic myeloid leukemia.
Adcetris (brentuximab vedotin) approved to treat 2 rare skin cancer: pcALCL and CD30 positive MF 
Ghassan Abou-Alfa, MD, medical oncologist at Memorial Sloan Kettering Cancer Center in New York City, specializes in gastrointestinal cancers.
Everybody else is figuring out what they want to do for a job, school, vacations. And I'm barely getting through every single day.
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