Last summer, my sister Becky approached me with an idea. She wanted my help building a platform at CNBC to spotlight the 30 million Americans living with a rare disease. Becky hoped that doing so would help give a voice to the millions of people that are often overlooked by the medical community … who…
Investors are concerned about the fates of multiple experimental drugs for hard-to-treat diseases following a string of recent rejections from the U.S. Food and Drug Administration. The FDA in the past year has denied or discouraged the applications of at least eight drugs, according to RTW Investments, including a gene therapy for Huntington’s disease from…
FILE PHOTO: The headquarters of the U.S. Food and Drug Administration (FDA) is seen in Silver Spring, Maryland November 4, 2009. Jason Reed | Reuters The Food and Drug Administration said Monday it has appointed former biotech executive George Tidmarsh as the agency’s top drug regulator. Tidmarsh, an adjunct professor of pediatrics and neonatology at…
Douglas Ingram, president and chief executive officer of Sarepta Therapeutics Inc., during the Forbes Healthcare Summit in New York, US, on Tuesday, Dec. 5, 2023. Michael Nagle | Bloomberg | Getty Images Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy treatment appeared at risk. The…
