Rare Disease Report
Patients & Caregivers


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.
Nasopharyngeal cancer has been linked to the Epstein-Barr virus.
The FDA granted orphan drug designation to tesevatinib for treatment of non-small cell lung cancer with epithelial growth factor receptor-activating mutations.
The FDA accepted a supplemental New Drug Application and granted Priority Review for Zelboraf, a therapy for Erdheim-Chester disease with a BRAF V600 mutation.
TheFDA has approved Imbruvica (ibrutinib) for treating adults with chronic graft versus host disease (cGVHD) after failure of one or more treatments.
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