Rare Disease Report


Bioverativ will collaborate with Bicycle Therapeutics to research, develop and commercialize sickle cell and hemophilia treatments.
To observe Pediatric Cancer Awareness Month, the WWE is partnering with the V Foundation through Connor’s Cure to raise funds for pediatric cancer research.
The Food and Drug Administration (FDA) has granted orphan drug designation to DCC-2618 for the treatment of glioblastoma multiforme and anaplastic astrocytoma.
This morning, the U.S. Food and Drug Administration (FDA) approved Pfizer’s Mylotarg (gemtuzumab ozogamicin) for the treatment of adults with newly diagnosed acute myeloid leukemia (AML).
Woodcock: We are communicating now to remind doctors and patients that Keytruda is not approved for treating multiple myeloma
Post-hoc analysis showed that Kyprolis + dex  increased overall survival by 7.6 months compared to Velcade + dex
On the same day that the first CAR-T-based gene therapy was approved, the FDA has approved a drug to help with a life-threatening side effect associated of it.
The U.S. Food and Drug Administration made history today when they approved the first-ever gene therapy, Kymriah (tisagenlecleucel), developed by Novartis.
Based on positive results from a Phase 3 head-to-head trial, the FDA and EMA have accepted the supplemental New Drug Application for Bosulif (bosutinib).
Lutathera, the 177-Lu somatostatin analog, is a new form of targeted therapy.
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