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.
Griffin Pitt, right, works with two other student researchers to test the conductivity, total dissolved solids, salinity, and temperature of water below a sand dam in Kenya.
Griffin Pitt’s upbringing made her passionate about water access and pollution, and Penn has given her the opportunity to explore these issues back home in North Carolina and abroad.
Helping robots work together to explore the Moon and Mars
Penn Engineers, NASA, and five other universities tested robotic systems designed to help unmanned explorers cooperate in the dunes of White Sands, New Mexico, paving the way for Moon and Mars exploration.
From framework to actions: Provost John L. Jackson Jr. talks Penn Forward
In a Q&A, Provost John L. Jackson Jr. explains the relationship between the strategic framework In Principle and Practice and Penn Forward—a new University-wide process and action plan that will advance Penn forward for the next decade and beyond.
