BOSTON, Mass. — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, announced today that the U.S. Food and Drug Administration (FDA) approved FILSUVEZ® (birch triterpenes) topical gel for the treatment of partial thickness wounds...
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CARLTON, Mercer County — Kyle Bennett is a 4-year-old, and he looks and behaves as such. It’s all shy smiles at first, then he opens up to show you what he’s into — kid stuff like his puppy, fishing with Dad, games, Scooby Doo … and more Scooby Doo. Kyle...
Denver, Colorado – Four children who acquired HIV in utero have remained free of detectable HIV for more than one year, according to new findings that were presented on March 6, at the Conference on Retroviruses and Opportunistic Infections (CROI) in Denver, Colo. The study is part of ongoing, multinational...
PHILADELPHIA, PA — Researchers at Children’s Hospital of Philadelphia (CHOP) developed a longitudinal atlas of neuroblastoma, a common and potentially deadly childhood cancer, to gain deeper understanding into precise molecular mechanisms underlying why and how certain treatments eventually become ineffective. The findings, which offer insights that could potentially lead to new personalized medicine approaches...
Philadelphia, PA – In a landmark study, an international consortium led by researchers at Children’s Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent...
Washington, DC — Children’s National Hospital and the Gilbert Family Foundation today announced a new $8 million, five-year grant to support groundbreaking research on neurofibromatosis type 1 (NF1). The grant, which is part of Gilbert Family Foundation’s Brain Tumor Initiative (BTI), will accelerate investigations aimed at improving the lives of...
INGELHEIM am RHEIN, Germany – China’s National Medical Products Administration (NMPA) has approved JASCAYD® (nerandomilast) as the first new treatment for adults with progressive pulmonary fibrosis (PPF) in more than five years, marking a breakthrough for millions affected by the life-threatening lung condition.4 The approval, based on the largest clinical trial program in PPF...
PARIS, France – The National Medical Products Administration (NMPA) in China has approved two innovative Sanofi medicines for rare hematologic diseases: Qfitlia (fitusiran) for hemophilia and Cablivi (caplacizumab) for acquired thrombotic thrombocytopenic purpura. These approvals mark another step in Sanofi’s long-term commitment to China, reinforcing the company’s ambition to bring...
Beijing, China – Colorectal cancer (CRC) is expected to contribute to a burden of 3.2 million new cases and 1.6 million deaths by 2040. CRC prevention strategies target lifestyle changes, screening high-risk individuals, and polyp removal, while the most effective treatment options are surgery and chemotherapy or radiotherapy. Although early...
Tang Dafeng began livestreaming on Douyin, the Chinese version of TikTok, at 10 pm, saying hello to the audience and introducing the story of her 3-year-old daughter, who two years ago was diagnosed with Rett syndrome, a rare disease that many people have never heard of. On Douyin, many mothers...