Tag Archives: gene

FDA blocks much-anticipated BioMarin hemophilia gene therapy

BioMarin Pharmaceutical shares plunged by a third after U.S. regulators rejected its potentially game-changing gene therapy for hemophilia A patients By LINDA A. JOHNSON AP Medical Writer August 19, 2020, 7:23 PM 4 min read Share to FacebookShare to TwitterEmail this article FAIRLESS HILLS, PA — Investors fled drug developer BioMarin in droves on Wednesday,… Read More »

Haunted by a Gene

Year after year for two decades, Nancy Wexler led medical teams into remote villages in Venezuela, where huge extended families lived in stilt houses on Lake Maracaibo and for generations, had suffered from a terrible hereditary disease that causes brain degeneration, disability and death. Neighbors shunned the sick, fearing they were contagious. “Doctors wouldn’t treat… Read More »

Six patients with rare blood disease are doing well after gene therapy clinical trial

Sarah C.P. Williams  UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people… Read More »

Researchers urge prostate cancer screening for men with BRCA gene defects

Prostate cancer screening with the prostate-specific antigen (PSA) test has been criticized for flagging too many slow-growing tumors that might never be life-threatening. But some men have inherited gene defects that boost their risk of developing prostate cancers that can be quite aggressive. Is PSA screening particularly well-suited for these genetically defined groups? New research… Read More »