Research & Development
Potential drug target to prevent small vessel disease identified in study
Scientists say lab-grown small blood vessels used in new disease models point to a theoretical potential treatment for a major cause of stroke and vascular dementia.
November 29, 2023
U.K. regulators approve "world-first" sickle-cell gene therapy
U.K. medical regulators have approved a “world-first” gene therapy that aims to cure two blood disorders: sickle cell disease and transfusion-dependent β-thalassemia. They are the first regulatory body to approve the treatment.
November 20, 2023
Networks of wearable sensors capture body sounds to improve patient monitoring
Researchers have developed soft, miniaturized wearable devices to continuously capture the sounds of breathing and other bodily functions.
November 16, 2023
U.K. trial approved for Hunter syndrome gene therapy
U.K. regulators have approved the “first ever” trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a rare lysosomal storage disorder.
November 9, 2023
Genomics England, Oxford Nanopore to collaborate on sequencing rare diseases
Genomics England and Oxford Nanopore Technologies have announced a strategic research partnership to resolve undiagnosed rare disease cases.
November 7, 2023
Study: Blood test improves bipolar disorder diagnosis through biomarkers
Diagnosis of bipolar disorder could be improved using a simple blood test to identify biomarkers associated with the condition, say researchers.
November 2, 2023
Oxford Nanopore partners with Fabric Genomics on assessment of genetic disease in children
Oxford Nanopore Technologies and Fabric Genomics have teamed up to provide a sequencing analysis platform for neonatal and pediatric intensive care units.
November 1, 2023
Vertex Pharmaceuticals, CRISPR Therapeutics gene-editing therapy to undergo FDA advisory committee review
An FDA advisory committee is scheduled to meet on October 31 to discuss and make recommendations on Vertex Pharmaceuticals' and CRISPR Therapeutics’ CRISPR-Cas9 gene-editing-based therapy, exagamglogene autotemcel.
October 30, 2023
Study: Proteomic profiling used to track effects of aging, diseases on the eyes
In a study published in
, researchers used proteomic profiling and artificial intelligence (AI) modeling to determine biomarkers and track the progression and effects of aging and disease on the eyes.
October 24, 2023
Researchers use motor sensors, machine learning to track Parkinson’s progression
Scientists say they have used data derived from sensors worn by patients and machine-learning analysis to accurately track the progression of Parkinson’s disease.
October 19, 2023
CMS opens new doors for cell and gene therapy under Medicaid
A new Medicaid-focused Innovation Center model encourages agreements between pharmaceutical companies and states for cell and gene therapies (CGT).
October 18, 2023
AI-driven method developed to identify therapeutic targets
Researchers have developed a method of identifying therapeutic targets for human diseases associated with protein phase separation that they say could be used to discover targets for Alzheimer’s and other neurogenerative diseases.
October 17, 2023
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