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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...
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...
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,...