Inherited Disease
News on Mendelian diseases, hereditary cancer, variant classification, cystic fibrosis testing and more.
In particular, patients who had fewer than 10 annual vaso-occlusive events before the gene therapy were more likely to have none after treatment.
In Brief This Week: Mount Sinai, AAVantgarde Bio, Lantern Pharma, Exegenesis Bio, AlveoGene
News items for the weeks of Nov. 25 and Dec. 2, 2024.
Vertex Pharmaceuticals, Bluebird Bio to Participate in CMS Cell and Gene Therapy Access Model
CMS has negotiated outcomes-based agreements for sickle cell gene therapies with the two companies on behalf of participating state Medicaid programs.
Biobanks have been making strides in gathering data from more diverse populations this year while the evidence for the clinical utility of embryo PRS testing remains insufficient.
Genetic Testing Challenges in Precision Medicine: Ataxia Patient Tested for Cardiomyopathy Genes
A neurologist orders the wrong genetic test for a patient experiencing peripheral neuropathy and doesn't realize it even upon seeing the report.