Originally published on January 25, 2021 11:40 am
Over the last several years, parents from across the country have appeared on television and news outlets to raise money to develop treatments for their children with rare genetic disorders.
Many of these families, including one from Kansas City, Missouri, have pinned their hopes on one Texas researcher, Dr. Steven Gray of the University of Texas Southwestern Medical Center in Dallas, who claims to be on the verge of treating a number of rare conditions. They ve raised millions of dollars to fund his research, although breakthroughs haven t happened to the extent many had hoped.
While gene therapy holds great promise, the growing trend of family-funded research concerns some medical ethicists, who say that suggestions to parents that treatments may be imminent can raise thorny ethical issues.
Cure SMA
Biogen Provides SMA Community Update on DEVOTE Study
Below we have provided an update from Biogen regarding the status of its DEVOTE clinical trial. For more information about SPINRAZA (nusinersen), visit https://www.curesma.org/spinraza/.
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Biogen’s DEVOTE study is designed to evaluate the safety, tolerability, and potential for even greater efficacy of SPINRAZA when administered at a higher dose than currently approved for the treatment of spinal muscular atrophy (SMA). The Phase 2/3 randomized, controlled, dose-escalating study will be conducted at approximately 60 sites in 26 countries around the world, and aims to enroll 152 individuals of all ages with SMA.
Famous Medical Researcher Dies After Receiving Wrong Drug; More medscape.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from medscape.com Daily Mail and Mail on Sunday newspapers.
December 18, 2020
COVID-19 has wreaked havoc around the globe, with more than 310,000 fatalities occurring in the United States alone. While much has been learned about the virus since it was first detected, the long-term effects on the health of coronavirus survivors may take years to understand. To find some answers, investigators from the Yale School of Medicine and partners across the country are launching a nationwide study of patients who were infected.
INSPIRE (Innovative Support for Patients with SARS COV-2 Infections Registry) is a Centers for Disease Control and Prevention-funded project led by Yale University with Rush University Medical Center; the University of Washington; the University of Texas Health Science Center at Houston; the University of Texas Southwestern Medical Center; the University of California, Los Angeles; the University of California, San Francisco; and Thomas Jefferson University. They will track 4,800 individuals to assess the longitudinal outcomes
Letters to the Editor Distribution of vaccines, sedition, Supreme Court dismissing Texas lawsuit, hypocritical rhetoric, book reviews
Readers worry whether vaccines will go to the correct people, explain what sedition means, justify the Supreme Court’s actions regarding the Texas lawsuit, are tired of false talk and hypocritical rhetoric and miss book reviews.
VP and Chief Pharmacy Officer Jon Albrecht receives a shipment of the Pfizer COVID-19 vaccine arriving at Methodist Dallas Medical Center in Dallas on Dec. 14, 2020. The hospital is one of four sites across Texas receiving the shipment today.(Lynda M. González / Staff Photographer)
Don’t cut in vaccine line