On Dec. 23, 2016 the U.S. Food and Drug Administration approved Biogen’s nusinersen (brand name Spinraza) for the treatment of spinal muscular atrophy (SMA), the leading genetic cause of death in infants.

The drug is designed to treat the underlying genetic defect in SMA, which means it could be particularly effective in slowing, stopping and perhaps reversing the symptoms of SMA.

“We’re extremely excited that nusinersen has been approved,” said Adrian Krainer, who led research to develop the drug at Cold Spring Harbor Laboratory in New York. “This is something we were hoping for … from the beginning when we started developing this therapeutic.”

In clinical trials to test Spinraza, participants who received treatment with the drug experienced life-changing outcomes — including the achievement of major motor milestones, such as the ability to roll over, sit unassisted and stand, that historically they wouldn’t have been expected to achieve.

“So we’re extremely pleased and proud, and looking forward to the impact it can have,” Krainer said.

This latest success for MDA’s research program is sure to inspire and motivate key players in the drug development arena to commit to developing treatments for neuromuscular diseases – leading to more treatments for the diseases under MDA’s umbrella.