International pharmaceutical company Roche, with its SMA drug development collaboration partners PTC Therapeutics and the SMA Foundation, said a clinical study called Moonfish (NCT02240355) that was investigating a compound and investigational medicine known as RG7800 for people with spinal muscular atrophy (SMA) was placed on clinical hold in…
Researchers from India reported the cases of two babies who were diagnosed with spinal muscular atrophy with respiratory distress syndrome (SMARD1).
Tetanus toxin may have a therapeutic role in spinal muscular atrophy (SMA) according to preliminary results published in the scientific journal Frontiers in Molecular Neuroscience.
Approximately one in 10,000 newborns are born with spinal muscular atrophy (SMA), a life threatening condition that results in muscle wasting, severely impaired mobility, and sometimes a shortened life span in severe cases. Currently, SMA is incurable but there are ways to manage the condition and bring comfort to patients. Learn how…
Screening for spinal muscular atrophy (SMA) in high-risk groups could greatly reduce the impact of the disease, according to a new article published by researchers in Ottawa. The report, “Opening the window: The case for carrier and perinatal screening for spinal muscular atrophy,” was published in the journal Neuromuscular Disorders.
Cure SMA-funded researcher Dr. Christian L. Lorson and his colleagues have published a paper in the journal Molecular Therapy describing a promising therapeutic prospect in a mouse model with severe spinal muscular atrophy (SMA). The article, “Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1…
Cure SMA has created a series of educational modules to address the topics, issues, and questions that are critical as the development of new drugs moves forward. The organization says a new stage has been reached in the efforts for U.S. FDA-approved treatments for spinal muscular atrophy (SMA),…
August, proclaimed SMA Awareness Month, is when advocacy and research organizations like CureSMA and the SMA Foundation and supporters seek to raise public consciousness of the genetic disorder spinal muscular atrophy (SMA). While the SMA support community works year-round to raise awareness of and funds for…
AveXis releases the interim results of their Phase 1 clinical trial testing the gene therapy drug AVXS-101 for the treatment of SMA.
A Phase 2 clinical trial investigating a potential treatment, CK-2127107, in people with spinal muscular atrophy (SMA), ages 12 years and older, is currently recruiting patients in the U.S. with varying degrees of disease severity (Types 2, 3, or 4). CK-2127107 is an investigational drug candidate designed for skeletal muscle and other debilitating neuromuscular…
