Rare Disease Report
Patients & Caregivers


Boston Biomedical has announced that the FDA has granted orphan drug designation to DSP-7888, a vaccine for the treatment of patients with brain cancer.
FDA granted Breakthrough Therapy Designation for Tagrisso (osimertinib) as a first-line treatment for metastatic EGFR+  NSCLC.
Trovagene, Inc. was granted Orphan Drug Designation from the U.S. Food and Drug Administration for its acute myeloid leukemia drug PCM-075.
Data from the GALLIUM Study that obinutuzumab-based immunochemotherapy and maintenance therapy is better for follicular lymphoma than rituximab-based therapy.
Patients with this rare lung cancer can now opt to orally ingest a 180 mg tablet
Clinical advancements in screening tests, surgical procedures, chemotherapy, etc are also helping to control this rare disease.
Pfizer announced plans for SpringWorks Therapeutics, which will be focused on just 4 experimental drugs, 3 of which are for rare diseases.
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.
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