MDUK is delighted to bring you the news that a highly complex, innovative gene therapy is to become available on the NHS in England for the treatment of spinal muscular atrophy (SMA) Type 1. Babies born with SMA Type 1, the most common form of the condition, are unable to...
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Garden City, NY — ProPhase Labs Inc. (NASDAQ: PRPH), a next-generation biotech, genomics, and diagnostics company, today reported its progress on the commercialization of its breakthrough BE-Smart test for esophageal cancer. The Company is aiming for commercial launch in the second half of 2024. Ted Karkus, CEO of ProPhase Labs,...
Patients with moderate or severe hemophilia A can benefit from early prophylactic antihemophilic factor (recombinant) (rAHF), according to an interim analysis presented at the 2021 British Society for Haematology annual meeting. The international AHEAD study is a 6 year non-interventional, prospective trial looking at joint health in patients receiving prophylactic...
The use of intravenous immunoglobulin (IVIG) may provide benefit against infections in subgroups of patients with multiple myeloma (MM), according to a retrospective, single-center crossover study that compared rates of infection in patients with MM during observation compared with those receiving IVIG. The study results were published in Clinical Lymphoma, Myeloma...
LEIDEN, Netherlands & CAMBRIDGE, Mass. — ProQR Therapeutics NV, a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced a collaboration with the Rett Syndrome Research Trust (RSRT) that will focus on the design and development of editing oligonucleotides...
LEIDEN, Netherlands & CAMBRIDGE, Mass. – ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), announced a global licensing and research collaboration with Eli Lilly and Company (Lilly) focused on the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The companies will use ProQR’s...
LEIDEN, Netherlands & CAMBRIDGE, Mass. – ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today announced results from a planned analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and...
WELLESLEY HILLS, Mass. — Joseph F. Finn, Jr., C.P.A. (“Finn”) announced today that Prospect Therapeutics has focused on developing GCS-100 for treatment of three blood-borne cancers: chronic lymphocytic leukemia (“CLL”), multiple myeloma (“MM”) and diffuse large B cell lymphoma (“DLBCL”). In clinical studies, GCS-100 has demonstrated activity as a single...
HERLEV, Denmark — A leading Danish cancer hospital has treated its first patient using RapidArc(R) radiotherapy after installing eight advanced radiotherapy treatment machines from Varian Medical Systems (NYSE: VAR) of Palo Alto, California. Herlev Hospital, to the north-west of Copenhagen, delivered the fast and efficient RapidArc treatment to a prostate...
A new prostate cancer risk assessment test, developed by a UCSF team, gives patients and their doctors a better way of gauging long-term risks and pinpointing high risk cases. According to UCSF study findings, published this week, the test proved accurate in predicting bone metastasis, prostate cancer-specific mortality, and all-cause...