Rare Disease Report
Patients & Caregivers


New Phase 1 clinical data for BLU-285 supports previous data that demonstrated the drug as exhibiting strong clinical activity and an encouraging safety profile.
Bristol-Myers Squibb announced that the FDA has expanded its approval of Sprycel tablets. The new indication includes children with rare chronic myeloid leukemia.
Adcetris (brentuximab vedotin) approved to treat 2 rare skin cancer: pcALCL and CD30 positive MF 
Ghassan Abou-Alfa, MD, medical oncologist at Memorial Sloan Kettering Cancer Center in New York City, specializes in gastrointestinal cancers.
Everybody else is figuring out what they want to do for a job, school, vacations. And I'm barely getting through every single day.
While hearing loss is just 1 of multiple side effects that many risk cancer survivors experience, this loss touches almost every aspect of one’s life.
Jill O'Donnell-Tormey, PhD, discusses immunotherapy and the immune system's role in rare cancers.
Rare Disease Report recently spoke with Jonathan Lim, M.D., the Chairman and CEO of Ignyta about the exciting results and next steps for Entrectinib.
The FDA announced the granting of regular approval to Alecensa (alectinib) for anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC).
Today, it was announced that Zelboraf received expanded approval for the drug to include the treatment of adult patients with Erdheim-Chester disease whose cancer cells have the BRAF V600 mutation.
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