Auckland, New Zealand – A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher. The patients from New Zealand, the Netherlands and the UK have hereditary angioedema, a genetic disorder characterised by severe, painful...
treatment News
MILAN, Italy and NEW YORK, NY — Genenta Science (NASDAQ: GNTA), a clinical-stage immuno-oncology (I/O) company developing a cell-based platform harnessing the power of hematopoietic stem cells to provide durable and safe treatments for solid tumors, today announced that: We have successfully dosed the first of three patients in Cohort...
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced the latest data from three pivotal Phase III studies of Venclexta® (venetoclax) – CLL14, MURANO and VIALE-A – to be presented at the European Hematology Association Virtual Congress, June 9-17 (EHA2021). Long-term follow-up data from the CLL14...
SOUTH SAN FRANCISCO, Calif. – Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that Evrysdi™ (risdiplam) data from the dose finding Part 1 of the pivotal FIREFISH study in infants with symptomatic Type 1 spinal muscular atrophy (SMA) were published in the February 24,...
Systemic sclerosis (SSc) is a rare disease that impacts up to 75,000 people in the United States Approximately 80% of SSc patients may be affected by interstitial lung disease (ILD), a progressive disease that can significantly impact lung function and can be life-threatening In a global study, Actemra reduced the...
SOUTH SAN FRANCISCO, Calif. — Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today data from the Phase III OCARINA II study (S31.006) of Ocrevus® (ocrelizumab), an investigational twice-yearly, 10-minute subcutaneous (SC) injection. Results showed near-complete suppression of clinical relapses and brain lesions in people...
These indigenous products will lead to drug prices for rare diseases being slashed up to 100 times of their current market value CHENNAI, India – Providing relief to patients with rare diseases across India, the Union Health Ministry has made available generic drugs to support the care and treatment of...
SARASOTA, Fla. and NOVATO, Calif. – GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced that they have received clearance from Health Canada to begin enrolling the Phase 1/2 study of GTX-102 in...
SHANGHAI, China — GenFleet Therapeutics, a clinical-stage biotechnology company developing cutting-edge therapies in oncology and immunology, announced that U.S. Food and Drug Administration (FDA) has recently granted another two designations for SLS009 (GFH009). GFH009 (highly selective CDK9 inhibitor) so far has received both fast track and orphan drug designations from the...
Exclusive rights for novel early-stage asset in cholangiocarcinoma have been acquired by GENFIT in these territories Phase 2 clinical program in cholangiocarcinoma expected to start the first half of 2022 Genoscience Pharma pursues the development with GNS561 in other oncology indications; will start phase 2 in hepatocarcinoma in 2022 Marseille,...