Rare Disease Report


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.
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.
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