Living with Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth disease (CMT) is one of the most common inherited neurological disorders, affecting approximately 1 in 2,500 people in the United States. The disease is named for the three physicians who first identified it in 1886 – Jean-Martin Charcot and...

FDA Grants Orphan Drug Designation for AML Treatment

Cantex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CX-01 for the treatment of acute myeloid leukemia (“AML”). CX-01 is an investigational agent that has the potential to enhance the...

Amit Rakhit, MD: Ovid Therapeutics’ Approach

Amit Rakhit, MD, MBA, Chief Medical and Portfolio Officer of Ovid Therapeutics, discusses his company’s approach in developing treatments for patients with rare neurologic disorders. Ovid Therapeutics is a biopharmaceutical company focused exclusively on...

Gamida Cell’s NAM Technology

Julian Adams discusses Gamida Cell’s NAM technology to develop two of its lead clinical programs by expanding blood forming stem and progenitor cells from umbilical cord blood to create highly functional, stand-alone grafts that serve as a universal bone marrow...
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