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,...
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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...
ENIGMA-TRS 1 is an international, one-year, double-blind, placebo-controlled Phase III study in at least 600 patients; enrollment to start imminently; 12-week study results expected in Q4 2026 ENIGMA-TRS 2, approved by the US Food and Drug Administration (FDA), is a US-based and international, 12-week, double-blind, placebo-controlled Phase III study in at...
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...
Los Angeles — Nexcella, Inc., a subsidiary of Immix Biopharma, Inc. (Nasdaq: IMMX) (“ImmixBio”, “Company”, “We” or “Us”) announced that NXC-201 treatment continues to demonstrate 100% complete hematologic responses and 100% organ response rate – cardiac, renal, liver response – in a total of 8 AL amyloidosis patients (an additional...
PHILADELPHIA, PA – A next-generation “armored” CAR T cell therapy showed promising results in a small study of patients whose B-cell lymphomas continued to resist multiple rounds of other cancer treatments, including commercially available CAR T cell therapies. The new therapy diminished cancer in 81 percent of patients and resulted...
NICE’s draft guidance published on 8 March 2021 recommends £1.79 million treatment Zolgensma (also called onasemnogene abeparvovec and made by Novartis Gene Therapies) for babies aged up to 12 months with type 1 SMA, This is one of the severest forms of the inherited condition and the life-expectancy of people with...
The British National Institute for Health and Care Excellence (NICE) has recommended Chiesi’s Elfabrio® (pegunigalsidase alfa) for Fabry disease (alpha-galactosidase deficiency) in adults. A new Fabry disease treatment The agreement made in the NICE’s Final Draft Guidance means there is now a “new treatment option for people living with Fabry...