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20 earlier
Decision expands options for eligible patients
Original title: “NHS backs delayed‑release treatment for cystinosis”
Study evaluates tri‑specific T cell engager LBL‑051
Original title: “Oblenio doses first patients in phase 1a autoimmune trial”
Designation supports accelerated access for investigational antibody
Original title: “BeOne Medicines secures UK innovation passport for HCC therapy”
EC expands use to protect youngest children
Original title: “Sanofi’s MenQuadfi approved for EU infants”
Positive two-year follow-up data for Otsuka Pharmaceuticals’ IgA nephropathy drug Voyxact could support the FDA’s ongoing review of Vertex Pharmaceuticals’ povetacicept, CEO Reshma Kewalramani said on the company’s second quarter earnings call.
Original title: “Vertex unfazed by competitive IgAN space as FDA verdict looms”
Harper Tanton will be the fourth child in the world to participate in the clinical trial for kids who have CTNNB1 syndrome. She largely depends on a wheelchair and has developmental and speech delays. The Tanton family travelled to Slovenia in 2024 to meet with the CTNNB1 Foundation and learn more about treatment options. (Submitted by Tara Tanton) The family eventually connected with the CTNNB1 Foundation, based in Ljubljana, Slovenia, which was founded and primarily funded by families from around the world. Researchers at the foundation are using gene replacement therapy to see if it can either stop the progression of CTNNB1 syndrome, or potentially cure it. Their clinical trial has been tested on two children so far. Harper Tanton and her mother will spend at least six months in Ljubljana, Slovenia, while Harper undergoes gene replacement therapy. (Carolyn Ray/CBC) The family of a Nova Scotia girl with an ultra-rare genetic disorder says they have hope for the first time that her condition will improve. Harper Tanton, who is eight years old, will be the fourth child in the world to participate in a clinical trial for kids who have CTNNB1 syndrome. She has an ultra-rare disease. Now this N.S. girl will be one of the first to test a possible cure | CBC News Loaded ... Harper Tanton will be the fourth child in the world to participate in the clinical trial for kids who have CTNNB1 syndrome. The foundation's website says because this is a first-in-human clinical trial, there may be unknown risks, and a child's condition could worsen.
Original title: “'We have to do it': Family of girl with ultra-rare disease prepares to test experimental treatment | CBC News”