New spinal muscle atrophy treatment gains approval
Zolgensma is based on a delivery system discovered by a Penn gene therapy pioneer, marking a new milestone in treating the rare disease.
The U.S. Food and Drug Administration recently approved a gene therapy known as Zolgensma for spinal muscle atrophy, the most common inherited fatal disease in infants.
The treatment, which is based on a decade of work by a team under the leadership of James Wilson, director of the University of Pennsylvania’s Gene Therapy Program and Orphan Disease Center, and a professor of medicine and pediatrics in the Perelman School of Medicine, corrects a gene mutation that eventually impedes a young child’s ability to walk, eat, and breathe.
Nanoparticle blueprints reveal path to smarter medicines
New research involving Penn Engineering shows detailed variation in lipid nanoparticle size, shape, and internal structure, and finds that such factors correlate with how well they deliver therapeutic cargo to a particular destination.
A generous gift from alumni Glenn and Amanda Fuhrman brings the work of internationally acclaimed artist Jaume Plensa to the University of Pennsylvania. The latest addition to the Penn Art Collection expands Philadelphia's public art.
A massive chunk of ice, a new laser, and new information on sea-level rise
For nearly a decade, Leigh Stearns and collaborators aimed a laser scanner system at Greenland’s Helheim Glacier. Their long-running survey reveals that Helheim’s massive calving events don’t behave the way scientists once thought, reframing how ice loss contributes to sea-level rise.
