Kara Elam,  —

Reldesemtiv improved muscle strength for walking and respiration in a dose-dependent manner in spinal muscular atrophy (SMA) patients, data from a Phase 2 clinical trial show. As a fast muscle activator, reldesemtiv will not treat the underlying causes of SMA — a lack of the SMN protein to nourish muscles…

The degeneration of spinal motor neurons in spinal muscular atrophy (SMA) may be the direct result of abnormal mitochondrial (energy source of cells) transport and morphology and subsequent reduced mitochondrial health. These are the conclusions of a study preformed by an international team of collaborative researchers led by scientists from the…

U.K. researchers funded by the SMA Trust have found that insufficient blood supply may well contribute to motor neuron loss in spinal muscular atrophy (SMA). The study entitled, “Vascular defects and spinal cord hypoxia in spinal muscular atrophy,” was published in the latest edition of the Annals of…

Dallas, Texas-based AveXis, Inc., a biotechnology company currently developing an experimental therapy for Spinal Muscular Atrophy (SMA) through the use of innovative gene therapy, recently announced that they have established a European Union (EU) based wholly-owned subsidiary called AveXis EU, Ltd.  This subsidiary will be used by AveXis to advance development of its…

A study published by a team of researchers at the Cold Spring Harbor Laboratory (CSHL) suggests that a drug to treat spinal muscular atrophy (SMA) in a mouse model is effective when administered subcutaneously in peripheral tissue and not just in the central nervous system (CNS). This important…