Rare Disease Report
Patients & Caregivers


Congenital Hyperinsulinism International (CHI) has its annual Sugar Soirée on Sunday, Nov 19, 2017 in NYC. Conan O'Brien will host. 
Sickle cell anemia or sickle cell disease is due to misshaped red blood cells.
The European Commission (EC) has approved the first-ever immunotherapy for the rare and aggressive skin cancer metastatic merkel cell carcinoma (mMCC).
The first patients have been dosed in a Phase 1/2 clinical trial of IMG-7289 for the treatment of myelofibrosis (MF), according to Imago BioSciences.
Sancilio Pharmaceuticals announced that the FDA has granted Rare Pediatric Disease designation to Altemia Soft Gelatin Capsules for sickle cell disease in children.
Jim Palma sits down with RDR to discuss cholangiocarcinoma, the disease with which Paul Poth, his brother-in-law and the founder of TargetCancer, was diagnosed.
Almost 3 years since its approval to treat patients with neuroendocrine tumors, Somatuline Depot has received a supplemental indication to treat carcinoid syndrome.
Accelerated Approval was granted to Bayer Healthcare Pharmaceuticals’ Aliqopa (copanlisib) today for the treatment of follicular lymphoma.
Amgen’s Mvasi (bevacizumab-awwb), a biosimilar to Avastin, is now FDA-approved for use in adults to treat multiple types of cancer.
Daiichi Sankyo and MD Anderson Cancer Center announced a multi-year collaboration dedicated to accelerating the development of novel therapies to treat AML.
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