Washington, DC – 2023 was a banner year for cell and gene therapy, with seven FDA approvals, according to the Alliance for Regenerative Medicine. And the pace doesn’t appear to be slowing at all as the second quarter of this year gets underway. The advocacy group is anticipating an even...
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WASHINGTON, DC — 60 Degrees Pharmaceuticals, Inc. , a pharmaceutical company focused on developing new medicines for infectious diseases, announced today the approval of an Investigational Review Board (IRB) sanctioned Phase IIA clinical study. The study aims to investigate the efficacy and safety of the ARAKODA® regimen of tafenoquine in...
SAN FRANCISCO, Calif. — 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) status to pegozafermin in patients with MASH. The...
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...
TORONTO, Canada – An international research team led by Krembil Brain Institute Neurologist and Senior Scientist, Dr. Anthony Lang, has proposed a new model for classifying Parkinson’s disease (PD). In recent decades, researchers have uncovered several biological factors that underlie PD. Key factors include a build up of the protein...
A prescription drug that helps Lore Wilkinson walk and talk despite a rare muscle disease cost her so little for more than a decade that she didn’t even use her insurance to pay for it. But now, her Medicare insurance is shelling out about $40,000 for a one-month supply of...
SAN DIEGO, Calif. – SYNC-T, an investigational therapy that combines a device-induced vaccination at the tumor site with intratumoral infusion of a multitarget biologic drug led to numerous clinical responses in patients with metastatic castration-resistant prostate cancer (mCRPC), according to results reported at the American Association for Cancer Research (AACR)...
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...