The designation is based on the results of the Phase II NOBILITY study that showed Gazyva, in combination with standard of care, helped more people achieve a complete renal response than standard of care alone Genentech today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to Gazyva® (obinutuzumab) for
The SanBio Group, a scientific leader in regenerative medicine for neurological disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) Designation for SB623 cell therapy for the treatment of chronic neurological motor deficits secondary to traumatic brain injury (TBI). The designation is based on clinical results
AACR Cancer Progress Report 2019 details 27 new cancer treatments, a record high number of U.S. cancer survivors, and the benefit of increasing federal investments in medical research through the National Institutes of Health and the National Cancer Institute The American Association for Cancer Research (AACR) today released its annual Cancer Progress Report highlighting how research largely
With wireless device, people without sight can detect motion, distinguish light and dark Source UCLA Health Seven years ago, Jason Esterhuizen was in a horrific car crash that destroyed his eyes, plunging him into total darkness. Today, he’s regained visual perception and more independence, thanks to an experimental device implanted in his brain by researchers
Researchers at Oxford University have discovered that a newly discovered group of cells can help repair tissues in the body. The researchers, who are supported by the NIHR Oxford Biomedical Research Centre, say these cells, which are abundant in our bodies, could be harnessed to help heal tissues and treat diseases such as infections of
The authors outline the latest discoveries in the biology of myelofibrosis (MF), discuss current clinical management of patients with MF, and summarize the ongoing clinical trials that hope to change the landscape of MF treatment. Myelofibrosis (MF) is a myeloproliferative neoplasm characterized by ineffective clonal hematopoiesis, splenomegaly, bone marrow fibrosis, and the propensity for transformation
Mark Zylka, PhD, in The UNC Neuroscience Center was awarded NIH funding for two 5-year projects, one jointly with Ben Philpot, PhD, to develop new knowledge with the potential to advance treatments for neurodevelopmental disorders. The National Institutes of Health have awarded two separate grants totaling $6.1 million to Mark Zylka, PhD, director of the
CRISPR Research Might Lead to Cure for Duchenne Muscular Dystrophy At this time, there is no cure for Duchenne muscular dystrophy (DMD), although there is one treatment for a subgroup of the disease. That is Sarepta Therapeutics’ Exondys 51 for DMD patients with a confirmed mutation amenable to exon 51 skipping. Recently the U.S. Food and
The collaborative project between the organizations will kick off at a launch meeting in September and will aim to reduce barriers for the development of new treatments and cures for rare diseases The Critical Path Institute (C-Path) and the National Organization for Rare Disorders® (NORD) will host a meeting on Tuesday, September 17 in Bethesda, MD
Applications and the limitations of real-world data. Source Contract Pharma There are approximately 7,000 distinct rare diseases that exist affecting 350 million people worldwide, and approximately 80% of those rare diseases are caused by faulty genes. Scientific advances such as the CRISPR/Cas9 genome-engineering system1 have simplified the pharmaceutical and biotech industry’s ability to develop gene therapies,
