News
First Patients Enrolled in First-of-Its-Kind International Trial Harnessing the Immune System Against Childhood Cancers
1+ day, 14+ hour ago (791+ words) WASHINGTON--(BUSINESS WIRE)--The first two patients have been enrolled in a new clinical trial taking place in the US and UK. Up to 60 eligible children and young people with cancer will receive a personalized investigational therapy that harnesses the…...
Encoded Therapeutics Announces FDA Alignment, Initiation of Pivotal Study, and ASGCT Presidential Symposium Presentation of ETX101 in Dravet Syndrome
2+ week, 1+ day ago (231+ words) Key meeting outcomes included: Dr. Rico added, "We are proud to also announce that initiation of both ENDEAVOR Part 1 B and Part 2 is already underway, demonstrating our ability to rapidly translate regulatory feedback into clinical execution. We expect patient dosing…...
Children's Hospital of Philadelphia Marks One-Year Anniversary of World's First Personalized CRISPR Gene Therapy for Child with Rare Genetic Disease
1+ mon, 1+ week ago (709+ words) Bio Space Children's Hospital of Philadelphia Marks One-Year Anniversary of World's First Personalized CRISPR Gene Therapy for Child with Rare Genetic Disease PHILADELPHIA, Feb. 25, 2026 /PRNewswire/ -- February 25, 2026 marks one year since KJ, an infant born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency,…...
Breakthrough T1 D Awards $1. 5 Million Grant to Blue Circle Health to Expand Access to Care for Underserved Adults with Type 1 Diabetes
2+ mon, 3+ week ago (166+ words) Grant will strengthen insurance and Medicaid navigation and continuity of care for adults living with T1 D "This grant allows us to reach more people, strengthen our services, and help individuals maintain access to care as their coverage or circumstances may…...
FDA Plans Black Box Warning for COVID-19 Vaccines: Report
3+ mon, 4+ week ago (250+ words) The FDA may be gearing up to slap a black box warning on COVID-19 vaccines, according to two individuals who spoke with CNN on Friday. A boxed warning, which alerts the public to the serious risks associated with a drug…...
CDC Highlights Efficacy of COVID-19 Shots in Kids Amid FDA Probe of Alleged Deaths
3+ mon, 4+ week ago (314+ words) i Stock, Eye Em Mobile Gmb H COVID-19 vaccines lowered the risk of emergency or urgent care visits associated with the disease by 76% in young children, as compared with those who were not immunized. Results showed that from 7 to 179 days…...
FDA Greenlights First Gene Therapy for Rare Pediatric Disease
3+ mon, 4+ week ago (252+ words) The FDA has approved the gene therapy etuvetidigene autotemcel for the treatment of Wiskott-Aldrich syndrome, a rare genetic disorder characterized by immune system abnormalities. The therapy will carry the brand name Waskyra. Waskyra is the first gene therapy for this…...
FDA Launches Probe Into Safety of Approved Anti-RSV Antibodies
3+ mon, 4+ week ago (222+ words) The FDA is looking into two approved antibodies used to prevent respiratory syncytial virus infections in babies, continuing its recent inquiry into the safety of pediatric preventative agents. Specifically, the agency is reviewing the safety of Sanofi and Astra Zeneca's…...
FDA's Prasad Links 10 Child Deaths to COVID-19 Vaccines, Eyes Stricter Review Guidelines
4+ mon, 1+ week ago (386+ words) Vinay Prasad, director of the FDA's Center for Biologics Evaluation and Research, said that "at least" 10 children have died "because of" COVID-19 shots, calling for changes to vaccine reviews and recommendations. Prasad made these claims in an internal memo made…...
Kennedy Revives Childhood Vaccines Safety Group, Led by NIH's Bhattacharya
7+ mon, 3+ week ago (315+ words) After nearly 30 years of dormancy, HHS is reinstating the Task Force on Safer Childhood Vaccines to develop and refine immunization guidelines for children across the country. The task force will submit a formal report to Congress within two years, with…...