Basel – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced new 2-year data from Part 2 of FIREFISH, a Phase 2/3 global study evaluating Evrysdi™ (risdiplam) in infants aged 1-7 months at enrollment with symptomatic Type 1 spinal muscular atrophy (SMA). The data showed Evrysdi continued to improve motor function between...
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HEBER CITY, Utah (ABC-4 Sports) – Just eight months ago, Jade Hafen wasn’t sure if he would be able to walk again. After competing in a calf roping event, Hafen collapsed, unable to move his legs. He was eventually diagnosed with Guillain-Barre syndrome, a dehabilitating nerve disorder in which the...
Catonsville, Maryland – Prostate cancer is the most common cancer in men other than skin cancer, and more than 288,000 new cases are diagnosed every year, according to the American Cancer Society. The disease’s fatality rate has decreased by more than half since the 1990s, but there is still room...
Cambridge, UK – Large-scale genetic analysis has helped researchers uncover the interplay between cancer-driving genetic mutations and inherited genetic variants in a rare type of blood cancer. Researchers from the Wellcome Sanger Institute, the University of Cambridge, and collaborators, combined various comprehensive data sets to understand the impact of both...
Paris, France – With ECRD 2024 scheduled for 15 and 16 May in Brussels and online, EURORDIS’ session, Revolutionising Funding Strategies for Breakthrough Therapies in Rarer Diseases, will address this critical issue. This session will not only highlight best practices but also showcase examples of therapies developed through innovative funding...
Dublin, Ireland – RCSI research has found that venetoclax, a medication currently approved for leukemia, has benefits for patients with multiple myeloma when used in combination with another drug. This discovery offers a new avenue of treatment options for the currently incurable disease. Multiple myeloma (MM) is a type of...
Manchester, UK – Babies and toddlers with a rare and fatal genetic condition can now receive life-saving treatment on the NHS for the first time. This follows clinical research studies first carried out at the National Institute for Health and Care Research (NIHR) Manchester Clinical Research Facility (CRF) at Royal...
CAMBRIDGE, Mass. & LEICESTER, England — RS Oncology (RSO), a clinical stage biotechnology company developing innovative therapies to eradicate mesothelioma and other diseases, today announced the successful dosing of the first patient in the Phase 2 dose expansion portion of its United Kingdom multicenter study (MITOPE; NCT05278975). Malignant pleural effusion...
SUNNYVALE, Calif. – Rubedo Life Sciences, a drug discovery company developing targeted therapeutics for age-related diseases, today announced a collaboration with Cedars-Sinai Medical Center in Los Angeles, to advance its idiopathic pulmonary fibrosis (IPF) program and establish collaborative studies. As part of the collaboration, Cory Hogaboam, Ph.D., Professor of Medicine...
New York — Rumi Scientific (“Rumi”), a developer of therapies for rare and neurodegenerative diseases discovered using its high throughput organoid phenotypic screening platform, announced the appointment of Allen Fienberg, Ph.D., as Chief Executive Officer (CEO) and Chairman of the Board of Directors. “Our human organoid-based high throughput drug discovery...