DUBLIN – Horizon Therapeutics plc (Nasdaq: HZNP) today announced that the U.S. Food and Drug Administration (FDA) has approved a prior approval supplement (PAS) to the previously approved Biologics Licensing Application (BLA) giving Horizon authorization to manufacture more TEPEZZA drug product resulting in an increased number of vials with each...
treatment News
New York, NY – A new study led by researchers at Hospital for Special Surgery (HSS) suggests that currently available therapies may help control chronic muscle inflammation in Duchenne muscular dystrophy (DMD), a severe condition that leads to muscle weakness and premature death. The study, published in PNAS, identifies new...
NEW YORK – Hoth Therapeutics, Inc. (NASDAQ: HOTH) a patient-focused clinical-stage biopharmaceutical company, announced it submitted an Orphan Drug Designation Request to the US Food and Drug Administration (FDA) for HT-KIT for the treatment of mastocytosis. HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frameshifting, resulting in the...
Mumbai, India – In November last year, Pratiksha Mane, 48, underwent surgery for Ewing’s Sarcoma, a rare form of breast cancer. She was diagnosed with cancer in her right breast, which was conserved post sugery by doctors at Mumbai’s Jaslok Hospital & Research Centre. As per the American Academy of...
San Francisco, CA – For Paul Bresge, getting into biotech wasn’t fulfilling a career goal. It was fulfilling a promise. One of his daughters, Tamar, was diagnosed with retinitis pigmentosa in 2010 at the age of 15, when the family was living in Toronto. “We were basically told—in the whole...
Sydney, Australia – UNSWmedical researcher Dr David Jacques and his team have discovered how the human immunodeficiency virus (HIV) breaches the cell nucleus to establish infection, a finding that has implications beyond HIV biology. To infect cells, HIV must enter the target cell and make its way to the nucleus...
LA JOLLA, CA —Pancreatic cancer is one of the deadliest cancers—only about one in eight patients survives five years after diagnosis. Those dismal statistics are in part due to the thick, nearly impenetrable wall of fibrosis, or scar tissue, that surrounds most pancreatic tumors and makes it hard for drugs...
SHANGHAI and CLINTON, N.J. — HuidaGene Therapeutics today announced the U.S. FDA granted Rare Pediatric Drug Designation (RPDD) to HG302, a novel CRISPR-Cas12 DNA-editing therapy, to treat Duchenne muscular dystrophy (DMD), affecting 1 in ~5,000 newborn boys. Unlike the in-vivo use of Cas9 system with limited genome-editing efficiency, high occurrence...
Human fibrinogen concentrate (HFC) has favorable pharmacokinetics for adult, adolescent, and pediatric patients with congenital fibrinogen deficiency (CFD), according to the results of open-label studies presented at the 2021 American Society of Pediatric Hematology/Oncology (ASPHO) meeting. The prospective studies FORMA-01, FORMA-02, and FORMA-04 looked at efficacy, safety, and dosing of...
ROCKVILLE, Md. — Human Genome Sciences, Inc. (Nasdaq: HGSI) today announced the presentation of results from a long-term Phase 2 continuation trial showing that BENLYSTA(TM) (belimumab, formerly LymphoStat-B(R)) was associated with sustained improvement in disease activity across multiple clinical measures, decreased frequency of disease flares, and was generally well tolerated...