Rare Disease Report
Patients & Caregivers


ArQule announced today that registrational trial of derazantinib (ARQ 087) in FGFR2 fusion driven second-line intrahepatic Cholangiocarcinoma, has initiated the dosing of patients.
The Pulmonary Hypertension Association is observing today, November 21, as Chronic Thromboembolic Pulmonary Hypertension (CTEPH) Awareness Day.
Tremeau Pharmaceuticals, Inc. was granted orphan drug designation this morning for TRM-201 (rofecoxib), intended for the treatment of hemophilic arthropathy.
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.
This morning, Akcea announced that all its marketing applications for volanesorsen have been accepted for review in the United States, Europe and Canada.
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.
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