American Society of Hematology Conference Coverage

This year’s American Society of Hematology (ASH) 2018 Annual Meeting was held on December 1-4 in San Diego, California. Physicians, researchers, and health care professionals learned about the latest advances in the treatment of blood cancers and blood...

FDA Approves First Treatment for Lambert-Eaton Myasthenic Syndrome

Some long-awaited good news arrived for the Lambert-Eaton Myasthenic syndrome (LEMS) community this week as the U.S. Food and Drug Administration approved Firdapse (amifampridine) tablets for the treatment of LEMS in adults. LEMS is a rare autoimmune disorder that...

FDA Approves Drug for Solid Tumors and NTRK Gene Fusions

The U.S. Food and Drug Administration granted accelerated approval to Vitrakvi (larotrectinib,  Loxo Oncology and Bayer) for the treatment of patients with solid tumors who have an NTRK gene fusion without a known resistance mutation. This approved indication applies...

Krabbe Disease

Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, New York City discusses Krabbe disease (also called globoid cell leukodystrophy), a degenerative disorder that affects the nervous system. It is caused by...

FDA Approves Venetoclax for Older Acute Myeloid Leukemia Patients

The FDA granted accelerated approval to Venetoclax (Venclexta) in combination with azacitidine, decitabine or low-dose cytarabine to treat newly diagnosed patients with acute myeloid leukemia (AML) aged 75 years or older. The approval also applies to adults with AML...

Leber’s Congenital Amaurosis Overview

  Daniel de Boer, Founder and CEO of ProQR, discusses Leber’s congenital amaurosis (LCA), the most common genetic cause of childhood blindness that affects about 15,000 patients in the Western world. LCA leads to poor vision and blindness for which there is...
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