Venture capital and angel investors are the lifeline of biotech companies. But what happens when obtaining an orphan drug designation no longer causes your stock price to double or triple in a matter of day, or allow you to do an IPO? Are VCs still interested? CheckOrphan rolled up its...
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Hepatocellular carcinoma (HCC) is the fifth most common malignancy worldwide and the third leading cause of cancer-related deaths. Type 2 diabetes mellitus has been associated with HCC. However, the relationship between type 2 diabetes mellitus and the underlying liver cirrhosis, and the effects of antidiabetic therapy on HCC risk have...
Rare diseases can’t wait. Rather than sit on the sidelines, many patients are taking the initiative to do what they can to accelerate the diagnostic process, improve understanding of their disease and spark research into potential therapies. The 2023 NEXT Report from nonprofit advocacy group, Global Genes, shares stories of...
Cologne, Gemany – High fat, low carb: A clinical study on special diets at the University of Cologne shows promising results on the use of a ketogenic diet as a possible treatment for hereditary polycystic kidney disease (ADPKD). This disease causes about 10 per cent of all cases of kidney...
Northwestern Medicine scientists have discovered a new biomarker to identify which patients with brain tumors called glioblastomas — the most common and malignant of primary brain tumors — might benefit from immunotherapy. The treatment could extend survival for an estimated 20% to 30% of patients. Currently, patients with glioblastoma do not...
WHITBY — It’s been only a year since Katie Sutherland lay in a hospital bed clinging to life, but the Whitby teen is getting ready to compete in an international sports competition. Katie, 16, underwent a double lung transplant last year and since then has been building her strength back...
Gene-targeted therapies have the potential to revolutionize medicine through treatments that modify or replace disease-causing genes with healthy variants. Gene therapy commonly involves using a modified virus to deliver the healthy genes into a patient’s cells. Once there, the new genetic material instructs the cell to produce the missing or...
LONDON – A world-first scientific study, published today in the New England Journal of Medicine, has shown that whole genome sequencing (WGS) can uncover new diagnoses for people across the broadest range of rare diseases investigated to date and could deliver enormous benefits across the NHS. The pilot study of rare...
NEW YORK — Incorporating whole-genome sequencing into healthcare around Stockholm has enabled more than 1,000 people with a rare disease to receive a molecular diagnosis, a new study has found. The Karolinska University Hospital and the Science for Life Laboratory (SciLifeLab) launched the Genomic Medicine Center Karolinska-Rare Disease (GMCK-RD), an...
NEW YORK — Whole-genome sequencing could serve as an alternative to cytogenetic analysis for acute myeloid leukemia or myelodysplastic syndromes, a new study has found. For AML and MDS, genetic abnormalities — typically chromosomal rearrangements — are used to classify patients into favorable, unfavorable, or intermediate risk groups, which then...