Rare Disease Report
Patients & Caregivers


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.
The companies are: Greenroads Health, Natural Alchemist, That's Natural!, Stanley Brothers
Accelerated approval was granted today by the FDA to Calquence (acalabrutinib) for the treatment of adults with mantle cell lymphoma.
This morning, Merrimack announced that orphan drug designation was granted to MM-121 (seribantumab) by the U.S. Food and Drug Administration (FDA).
Updated results from an ongoing Phase 1/2 study of X4P-001-IO in combination with Inlyta to treat patients with clear cell renal cell carcinoma were announced.
The large phase 3 study observed that efficacy was the same for both treatments.
Rare Disease Report spoke with Steven Benner, MD about the new designation, the development program, and the functionality and target for the potential therapy.
A new dosing regimen for Amgen’s multiple myeloma drug Kyprolis (carfilzomib) appears to be more effective than what is currently approved.
Data concludes that while NETs in the distal of the small bowel are larger than in the middle, there are no differences in progression-free or overall survival
At the NANETS Annual Symposium results from a retrospective database analysis of somatostatin analog (SSA) usage in neuroendocrine tumors (NETs) were shared.
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