Rare Disease Report
Patients & Caregivers


A Phase 1 clinical study testing CAR T-cell therapy, UCART123,to treat blastic plasmacytoid dendritic cell neoplasm at the MD Anderson Cancer Center has begun.
Curtana announced the FDA has granted CT-179 with an Orphan Drug Designation to treat for gliomas, including glioblastoma in adults and brain tumors in children.
The FDA approved olaparib tablets (Lynparza) for adult patients with recurrent epithelial ovarian fallopian tube, or primary peritoneal cancer.
FDA has approved Besponsa (inotuzumab ozogamicin) for treating adults with relapsed or refractory B-cell precursor ALL
NF-1 patients often suffer from chronic constipation that can greatly impact their health and quality of life.
Singapore-based clinical-stage biotechnology company Lion TCR was granted 2 orphan drug designations today by the U.S. Food and Drug Administration (FDA) for T-cell therapies.
Interim analysis of a Phase 2/3 study testing CA4P in platinum-resistant ovarian cancer patients is struggling to meet its primary endpoint.
Head and neck cancers begin in the mouth, nose, throat, larynx, sinuses, or salivary glands. Most are squamous cell carcinomas.
H3B-8800, was granted orphan drug designation by the FDA for the treatment of patients with acute myeloid leukemia, and chronic myelomonocytic leukemia.
Mariann's plexiform fibrohistiocytic sarcoma had taken over most of her back. Her treatment required a donor that was compatible.
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