Boston, Massachusetts – Cerebral adrenoleukodystrophy (CALD) is a rare progressive, genetic brain disease that primarily presents in young boys, causing loss of neurological function and ultimately leading to early death. Researchers from Massachusetts General Hospital, a founding member of the Mass General Brigham healthcare system, Boston Children’s Hospital, and collaborators...
Latest News
University of Florida researchers have come up with a new gene therapy method to disrupt cancer growth by using a synthetic protein to induce blood clotting that cuts off a tumor’s blood and nutrient supply. In mice implanted with human colorectal cancer cells, tumor volume decreased 53 percent and cancer...
Bethesda, MD – In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range of bodily systems, leading to severe cardiac, neurological, and psychiatric symptoms as well as physical differences such as webbed...
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...
Stamford, Conn. — GeneDx Holdings Corp. (Nasdaq: WGS) (“GeneDx” or the “Company”), a leader in delivering improved health outcomes through genomic and clinical insights, reported its financial results for the fourth quarter and full year ended December 31, 2022. “Entering 2023 with a new corporate strategy and a focus on...
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...