During the last week of December 2016, the FDA formally approved a new drug from Biogen called Spinraza (nusinersen) for the treatment of spinal muscular atrophy (SMA.) In SMA, very little of a specific protein is made at full length. Instead, most of that protein is in a short, unstable form. Without the protein, loss of motor neurons from the spinal cord occurs, with resulting weakness and atrophy of the voluntary muscles. In the severest forms of the disease, premature death may occur because of failure of the respiratory muscles.
Infants with type 1 SMA can’t sit or hold their heads up, and progressively lose what movement ability they have. These babies typically don’t live past the age of 1 year, with death often due to respiratory failure unless the infant is placed on a ventilator. Other, milder forms of SMA (types 2, 3 and 4) involve difficulties with movement and are associated with a normal or somewhat reduced lifespan.
Just five years ago, there were no approved therapies to treat the underlying cause of SMA. MDA has funded foundational work in SMA and invested nearly $750,000 in awards for early-stage development of nusinersen. Since its inception, MDA has invested more than $45 million in SMA research. This breakthrough will help Nebraskans like Ollie, pictured, and is only possible because of your gifts.
Read more about Spinraza here.