Basel – Novartis today announced new data that reinforce the transformational benefit of Zolgensma® (onasemnogene abeparvovec), an essential one-time treatment for spinal muscular atrophy (SMA). The overall safety profile remains favorable following presymptomatic treatment, in the long-term follow-up period from clinical studies and in the real-world setting. These data were...
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Basel – Novartis today announced data that reinforce the transformational benefit of Zolgensma® (onasemnogene abeparvovec), an essential, one-time treatment and the only gene therapy for spinal muscular atrophy (SMA). New late-breaker data from the completed two-copy cohort of the Phase 3 SPR1NT clinical trial demonstrate age-appropriate milestone development in presymptomatic children...
Chapel Hill, North Carolina – UNC Health is now enrolling patients as part of a multicenter clinical trial for patients who have been newly diagnosed with glioblastoma (GBM). The phase 2b clinical study administers IGV-001, a combination immunotherapy developed by biotech company Imvax. UNC Health is one of multiple academic...
ZURICH, Switzerland – Fatigue is common in people with inflammatory rheumatic and musculoskeletal diseases (RMD). The causes of fatigue are not well understood, and it is likely that they vary between people and over time. From a patient perspective, fatigue has a significant and detrimental impact on daily life, and...
NAARDEN, Netherlands and MIAMI, Florida — NewAmsterdam Pharma Company N.V., a late-stage, clinical biopharmaceutical company developing oral, non-statin medicines for patients at risk of cardiovascular disease (CVD) with elevated low-density lipoprotein cholesterol (LDL-C), for whom existing therapies are not sufficiently effective or well-tolerated, today announced positive topline data from the...
INDIANAPOLIS, Indiana — A new study led by Indiana University School of Medicine researchers found a brain network condition called “explosive synchronization” could be the cause of extreme pain crises in people with sickle cell disease. “These findings could have a significant impact on patients with this disease,” said Ying Wang,...
LOS ANGELES, Calif. – A previously unidentified genetic mutation in a small protein provides significant protection against Parkinson’s disease and offers a new direction for exploring potential treatments, according to a new USC Leonard Davis School of Gerontology study. The variant, located in a mitochondrial microprotein dubbed SHLP2, was found...
UNIVERSITY PARK, Pa. — For some people, extreme stressors like psychiatric disorders or childhood neglect and abuse can lead to a range of health problems later in life, including depression, anxiety and cardiovascular disease. A new study led by researchers in the Penn State Center for Healthy Aging identified genetic indicators that...
London, UK – More than 200 genes linked to depression have been newly identified in a worldwide study led by UCL researchers. The research, published in Nature Genetics, found more than 50 new genetic loci (a locus is a specific position on a chromosome) and 205 novel genes that are...
MILAN, Italy — Newron Pharmaceuticals S.p.A., a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system, today announced the completion of patient enrollment in study 008A, its potentially pivotal study with evenamide in patients suffering from schizophrenia. Study 008A...