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.  

The experimental drug RG3039, being developed by Repligen Corp. of Waltham, Mass., as a potential treatment for spinal muscular atrophy (SMA), has moved into its next phase of testing.

RG3039 previously was tested in a phase 1a trial, in which healthy adult volunteers received a single dose of the drug. Now, in a phase 1b trial, healthy volunteers will receive multiple doses of the drug.

In 2003, the average life expectancy for a person with the genetic lung disease cystic fibrosis was 33 years. But at some cystic fibrosis medical centers, life expectancy was more than 47 years. The Cystic Fibrosis Foundation wanted to know why. Using its national patient registry, the foundation identified which medical centers achieved optimum outcomes, identified the clinical practices that led to those outcomes and then disseminated that information to its national clinical network — improving care for everyone with cystic fibrosis.

The National Institute of Neurological Disorders and Stroke (NINDS) has launched a study to look at natural history (general disease course) and search for disease indicators called biomarkers in infants with type 1 spinal muscular atrophy (SMA).