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...
treatment News
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...
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...
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...
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...