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New Treatment Options for Hemoglobin-Based Blood Disorders

    Sickle cell disease is a group of inherited disorders that affects hemoglobin, the oxygen-carrying protein in red blood cells (RBCs), and causes these cells to distort in shape at low oxygen levels. This condition affects 1 in 500 Americans of African descent and 1 in 1,000-1,400 of Hispanic descent, as well as millions...

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Long Awaited CMS Rules on Medicare Coverage for CAR-T Therapies

In August, the Centers for Medicare and Medicaid Services (CMS) finalized long-sought rules framing the coverage of chimeric antigen receptor T-cell (CAR-T) treatments. Two CAR-T therapeutics are currently on the market: Gilead’s Yescarta and Novartis’ Kymriah, both approved for diffuse large B-cell lymphoma (DLBCL) with the latter also approved for acute lymphocytic leukemia (ALL). Under...

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New Strategies Against a Hard-to-Treat Killer — Pancreatic Cancer

Pancreatic cancer was the third leading cause of cancer-related death in the United States in 2018. This year, experts estimate that 56,770 new cases will be diagnosed and 45,750 deaths will be recorded. Pancreatic cancer is especially lethal because in its early stages, when the disease is easiest to treat, patients are usually asymptomatic. The disease...

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New Approvals for Tumor-Agnostic Cancer Treatments — and the Questions They Raise

On August 15, the U.S. Food and Drug Administration granted accelerated approval to Genentech/Roche’s Rozlytrek (entrectinib) for the treatment of adult and adolescent patients with solid tumors that exhibit neurotrophic tyrosine receptor kinase (NTRK) fusions and for whom there are no existing effective treatments. While NTRK fusions are rare, appearing in about 1% of solid...

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The Missing Piece in the Brexit Prep Jigsaw

After three years since the United Kingdom’s vote to leave the European Union and a number of extensions, the country is fast approaching the latest deadline of October 31, 2019. The new Prime Minister, Boris Johnson, and the leaders of the EU have been at loggerheads, and the possibility of a deal or no deal...

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Focusing on NASH

Non-alcoholic fatty liver disease (NAFLD), the build-up of fat in the liver that is not caused by alcohol, affects almost 100 million people in the United States alone. With no drug therapies available for the condition, treatment has focused mostly on weight loss. The more severe form of NAFLD, non-alcoholic steatohepatitis (NASH), causes the liver...