Novartis today presented new data that continue to support the clinical benefits of Zolgensma® (onasemnogene abeparvovec), the only one-time gene therapy for the treatment of spinal muscular atrophy (SMA). Final data from the SMART study highlight the safety and efficacy profile of Zolgensma in children with SMA weighing ≥ 8.5 kg to ≤ 21 kg, with a mean age of 4.69 years, most of whom (21/24, 87.5%) had discontinued use of another disease modifying therapy at the time of treatment.

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Cleveland Clinic researchers have discovered a key mechanism used by Kaposi's sarcoma-associated herpesvirus (KSHV), also known as human herpesvirus 8 (HHV8), to induce cancer. The research points to effective new treatment options for KSHV-associated cancers, including Kaposi's sarcoma, primary effusion lymphoma, and HHV8-associated multicentric Castleman disease.

Pfizer Inc. (NYSE: PFE) today announced top-line ABRYSVO® vaccine efficacy and safety data for respiratory syncytial virus (RSV) in adults 60 years of age and older following a second season in the Northern and Southern Hemispheres from the ongoing pivotal Phase 3 clinical trial (NCT05035212) RENOIR (RSV vaccine Efficacy study iNOlder adults Immunized against RSV disease).

A study by the Institut de Neurociències of the UAB (INc-UAB) demonstrates in animal models the benefits of vorinostat after having suffered a stroke. The drug, used in humans to treat cutaneous T-cell lymphoma, has been proved to mitigate brain injuries and help in restoring brain tissue.

Ischemic stroke is the second leading cause of death worldwide and occurs when blood flow cannot reach the brain due to an obstruction.

Bayer announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for BAY 2927088 for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have received a prior systemic therapy. BAY 2927088 is an oral, reversible tyrosine kinase inhibitor (TKI) that potently inhibits mutant human epidermal growth factor receptors 2 (HER2),

Lung cancer is not the most common form of cancer, but it is by far the deadliest.

Despite treatments such as surgery, radiation therapy and chemotherapy, only about a quarter of all people with the disease will live more than five years after diagnosis, and lung cancer kills more than 1.8 million people worldwide each year, according to the World Health Organization.

Pfizer Inc. (NYSE: PFE) announced that the European Commission (EC) has granted marketing authorization for VELSIPITY® (etrasimod) in the European Union to treat patients 16 years of age and older with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response, or were intolerant to either conventional therapy, or a biological agent.

University of North Carolina at Chapel Hill researchers have developed a new drug delivery platform that harnesses helical amyloid fibers designed to untwist and release drugs in response to body temperatures.

A new research paper published on Jan. 26 in Nature Communications reveals groundbreaking structural details into how diseases form much like Alzheimer's disease.

Before orally administered drugs can make their way throughout the body, they must first bind to membrane proteins called drug transporters, which carry compounds across the intestinal tract and help them reach their intended targets. But because one drug can bind to several different drug transporters, they may struggle to get past this gut barrier, potentially leading to decreased drug absorption and efficacy.