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Bloomington, MN – Pandemic-era changes may have permanently altered the clinical trial landscape as decentralized clinical trials gain broader acceptance. During COVID-19, more than three-quarters (76%) of pharmaceutical companies, device manufacturers and contract research organizations conducted off-site clinical trials. These types of trials specifically provide increased opportunity for individuals with...
Singapore — Just nine Singaporeans – or about 0.4 per cent of the 2,000 to 3,000 who have chronic rare diseases here – have received help from the Rare Disease Fund (RDF) since it was started in 2019. Most patients with rare diseases are able to get financial help from...
San Francisco, Calif. –  Multiple sclerosis patients whose blood tests reveal elevated NfL, a biomarker of nerve damage, could see worsening disability one to two years later, according to a new study spearheaded by researchers at UC San Francisco. The study is the first to quantify the timeframe preceding disability...
Checkpoint with forkhead and ring finger (CHFR) is a mitotic stress checkpoint gene whose promoter is frequently methylated in various kinds of cancer. In gastric cancer, CHFR promoter hypermethylation has been reported to lead to chromosome instability (CIN) and genetic instability is one of the hallmarks of human cancer. A...
Toruń, Poland – Boys suffering from Barth syndrome, a genetic determined disease with a very high mortality rate, finally have hope for a cure. What has contributed to this finding is research of Dr. habil Karolina Mikulska-Ruminska, Nicolaus Copernicus University (Torun, Poland) CU Professor from the Institute of Physics. The...
Two biotechs are shoring up their pipelines and commercial aspirations, tacking on a clinical-stage rare disease drug and a preclinical cancer med, respectively. Fortress Biotech-founded Avenue Therapeutics is adding on a phase 1/2-stage asset from AnnJi Pharmaceutical for the treatment of spinal and bulbar muscular atrophy (SBMA). Financial details are...