Spinal Muscular Atrophy (SMA)

A one-hour, MDA-sponsored webinar features two physicians and the parent of a child with spinal muscular atrophy (SMA) who has undergone bracing and surgery for a spinal curvature, as well as questions and answers from listeners.

The biotechnology firm Repligen today announced it has entered into an agreement with the global pharmaceutical company Pfizer to advance Repligen’s spinal muscular atrophy (SMA) program, which includes RG3039, an experimental drug for SMA whose development MDA has supported.

A small-molecule drug candidate for spinal muscular atrophy (SMA) and an effective strategy for advancing therapy development are the primary outcomes being reported by the Spinal Muscular Atrophy (SMA) Project, a program established in 2003 by the National Institute of Neurological Disorders and Stroke (NINDS) to accelerate the development of therapeutic candidates for thi

Like many high school seniors, Drew is busy choosing a college and preparing for the big transition from life at home to life on a college campus. Unlike his peers, Drew has Duchenne muscular dystrophy.

A phase 1b/2a trial to test the safety and tolerability of multiple doses of the experimental drug ISIS-SMNRx in children with spinal muscular atrophy (SMA) is now open at sites in New York and Salt Lake City, with additional sites expected to open in Boston and Dallas.

Investigators hope to enroll a total of 24 children with SMA ages 2 to 15 in the trial.

Investigators at Northwestern University in Chicago are seeking people with spinal muscular atrophy (SMA), ages 2 to 21, to participate in a study about preferences and involvement in recreational and leisure activities.  

Results from a study in fruit flies conducted by scientists in the Motor Neuron Center at Columbia University Medical Center in New York suggest that spinal muscular atrophy (SMA)— commonly thought to be a disease of muscle-controlling nerve cells called motor neurons— instead results from the dysfunction of motor circuits (networks made up of different types of specialized neurons that coordinate muscle movement).