Rockville, MD – According to several key opinion leaders (KOLs) interviewed by GlobalData, pipeline gene therapies hold the greatest potential to transform the Gaucher disease landscape. While the importance of these novel therapies has been emphasised, there remains a significant amount of groundwork to be covered before these therapies receive...
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An expert panel failed to reach consensus on how to treat some patients with severe hemophilia A, suggesting that further study is needed where the optimal treatment remains equivocal, according to research published in the Journal of Blood Medicine. Hemophilia A is linked with a reduced quality of life (QoL)...
By Raffaele Pereno – PhD, MBA BASEL, Switzerland – Reporting from the Festival of Biologics 2025, CheckOrphan attended a thought-provoking keynote panel featuring Dr. Marco Traub, Founder of the Trans European Stem Cell Therapy Society (TESCT), and Dr. Pedro Cruz, Chief Technical Operations Officer at iBET – Instituto de Biologia...
New York, New York – Because bleeding from the reproductive tract is a naturally occurring event during menstruation and childbirth, women who exhibit menorrhagia, or excessive bleeding after their menstrual cycle, may have underlying diseases that are underdiagnosed. In order to address important issues related to the diagnosis and management...
Los Angeles, Calif. – In one of the largest ever gene-environment interaction studies of red meat and colorectal cancer, which explored the impact of red meat consumption on a person’s cancer risk based on their genotype, researchers have identified two genetic markers that may help explain the association between the...
Norman, Oklahoma – People who are diagnosed with head and neck cancer often receive a standard type of chemotherapy as part of their treatment. If they are exposed to secondhand smoke during chemotherapy — even if they have never smoked themselves — the treatment may be far less effective at...
ATLANTA, Georgia — Expression Therapeutics, Inc., a clinical-stage biopharmaceutical company developing a curative autologous stem cell therapy for hemophilia A, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation and Rare Pediatric Disease Designation to its investigational therapy. The dual designations recognize the severity and...
Extended induction chemotherapy with topotecan, cyclophosphamide, and etoposide does not improve event-free survival (EFS) and overall survival (OS) in patients with high-risk neuroblastoma, and it increases the number of toxic side effects, according to findings published in the Annals of Oncology. The open-label, prospective, randomized controlled phase 3 NB2004-HR trial (ClinicalTrials.gov...
London, UK – Prolonged use of certain progestogen hormone drugs is associated with an increased risk of developing a type of brain tumour known as an intracranial meningioma, finds a study from France published by The BMJ today. The researchers say this study is the first to assess the risk associated with...
Extensive examination of lymph nodes and node stations appeared to be crucial to predicting accurate staging and survival outcomes in patients with pure-solid, non-small cell lung cancer (NSCLC) who underwent lobectomy and ipsilateral lymphadenectomy, according to the results of a study published in the Journal of the National Comprehensive Cancer Network....