MDA-funded researchers say SMN, which is deficient in spinal muscular atrophy, is most needed during early life and in skeletal muscle and nerve tissues
Raising levels of the SMN protein, which is deficient in patients with the most common form of spinal muscular atrophy (SMA), has been the holy grail of therapy for this disease since the 1990s and is the goal of several experimental compounds now in development.
The quest to cure spinal muscular atrophy still has many years to go, but the signs of progress are unmistakable
A Closer Look: SMA Slideshow
August is often referred to as Spinal Muscular Atrophy (SMA) Awareness Month, but for MDA, work on finding treatments for this degenerative disease — and providing assistance to individuals and families affected by it — is a year-round event.
Today, research aimed at slowing, stopping or even preventing spinal muscular atrophy (SMA) is advancing with more speed...
Researchers look at the 'window of opportunity' for effective treatment in spinal muscular atrophy and the therapeutic potential of celecoxib
Recent spinal muscular atrophy (SMA) research includes findings that shed additional light on the optimal "window of opportunity" for treatment and point toward a potential candidate for therapeutic development.
The trial is designed to evaluate the safety of multiple doses of RG3039 in healthy volunteers and to determine pharmacokinetics (the way the body affects a drug)
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.
Reversing muscle damage caused by low SMN; nerve-muscle communication breakdown; phase 2 trial of olesoxime now fully enrolled
Skeletal muscle damage reversed in SMA mice
In addition to muscle damage caused by the loss of motor neurons in the spinal cord, skeletal muscle degeneration in spinal muscular atrophy (SMA) also stems from low levels of the protein SMN in muscle fibers. Now a team of scientists from Germany and the United Kingdom reports that muscle damage caused by low SMN can be reversed with drugs.
Treatment with the protein prolactin improved motor function and enhanced survival time in mice with a disease resembling severe SMA
The administration of a protein called prolactin has been shown to slow weight loss, improve motor function and increase life span by approximately 70 percent in mice with a disease resembling a severe form of spinal muscular atrophy (SMA).
Prolactin is a hormone whose primary function is to promote milk production in women who are breastfeeding.
MDA-supported scientists found raising SMN protein levels conferred significant benefits on young SMA mice, even after symptom onset
The "window of opportunity" for treating infants at risk of developing spinal muscular atrophy (SMA) may not be as narrow as some experts have feared, new experiments in mice suggest.