Spinal Muscular Atrophy (SMA)

SMA — John Manfredi, Ph.D.

John Manfredi, chief scientific officer at Sfida BioLogic in Salt Lake City, Utah, was awarded an MDA research grant totaling $161,995 over a period of two years to study the potential of new compounds for treatment of spinal muscular atrophy (SMA). The new grant complements previous MDA-funded research by Manfredi into potential therapeutics for SMA.

MDA Webinar Discusses Spinal Bracing and Surgery in Children

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.

SMA — Christine DiDonato, Ph.D.

Christine DiDonato, assistant professor of pediatrics at Northwestern University in Chicago, Ill., was awarded an MDA research grant totaling $405,000 over a period of three years to test treatment strategies for spinal muscular atrophy (SMA).

SMA — Gary Bassell, Ph.D.

Gary Bassell, professor of cell biology and neurology at the Emory University School of Medicine in Atlanta, Ga., was awarded an MDA research grant totaling $405,000 over a period of three years to discover new functions of the SMN protein in spinal muscular atrophy (SMA).

Federal SMA Project Issues Progress Report

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

Ronald Abbott

Full name: 
Mr. Ronald Abbott
Ronald Abbott
Spinal Muscular Atrophy (SMA)

MDA Building ‘Transitional Freedom’ Partnerships

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.

Trial of Antisense Drug Opens for Children with SMA

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.

SMA: Participants Sought for Study About Recreational Activities

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.  

Dysfunction of Motor Circuits May Underlie SMA

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).