Rare Disease Report
Patients & Caregivers


Caroline has profound hearing loss as a result of the chemotherapy she needed to combat the neuroblastoma she had as a child.
While he was once a gregarious, out-going boy, he is now lonely and frustrated because he can not keep up with the conversations.
Numerous social and psychological problems can stem from a little hearing loss that can profoundly impact a child growing up.
Rare Disease Report spoke with Dr. Rajput about her research that displays the profound impact that hearing loss had on the quality of life.

Until recently, the only option was to accept hearing loss a side effect of being cured of cancer. New options are becoming available.
October is Liver Cancer Awareness month, and the Fibrolamellar Cancer Foundation is making sure the world's fifth most common cancer type is properly recognized.
This morning, Astellas announced that it was granted Fast Track designation for the development of gilternitinb in adult patients with AML.
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.
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