[Editor’s Note:  This is part of a series of articles into the discovery and development of Evrysdi, SMA’s newly approved disease-modifying therapy and its first oral and at-home one, as well as the scope of SMA issues and treatments. Here, we talk with scientists about how this…

Evrysdi (risdiplam) is an oral liquid treatment approved in the U.S. for infants through adults with spinal muscular atrophy (SMA) — patients age 2 months and older with any disease type. Roche and Genentech developed Evrysdi in collaboration with PTC Therapeutics and the SMA…

The U.S. Food and Drug Administration (FDA) has approved Evrysdi — formerly known as risdiplam — as the first oral and at-home treatment for adults, children, and infants 2 months of age and older with all types of spinal muscular atrophy (SMA). With this action, Evrysdi — marketed by…

It’s probably safe to say that these past four months have been a blur for many. It seems that mere blinks ago it was mid-March, the last days of normalcy in untold routines, and the beginning of upheavals that have defied description. Somehow, it’s already four months…

The Iowa Department of Public Health has begun a pilot effort to screen newborns for spinal muscular atrophy (SMA). Babies born in Iowa are now being screened for more than 40 inherited disorders, with a goal of identifying conditions that, if caught early, can be treated or cured.

Asuragen’s diagnostic test for the analysis of genes associated with spinal muscular atrophy (SMA) — called AmplideX SMA Plus Kit — was given the European CE mark, approving it for commercial use, the company announced. The designation means the AmplideX kit complies with the European…

The Illinois Department of Public Health (IDPH) now includes spinal muscular atrophy (SMA) as part of its newborn screening effort. The addition of SMA — the 49th disorder in the state’s screening program — means that every baby born in Illinois will be tested for the autosomal recessive…

Levels of the survival motor neuron (SMN) protein in the blood of people with spinal muscular atrophy (SMA) shows promise as a biomarker for predicting disease severity, a study reports. Specifically, SMN levels correlate with the severity of motor-neuron loss — denervation — and with the number of…

A baby with spinal muscular atrophy (SMA) type 0 treated with both Spinraza (nusinersen) and Zolgensma is showing benefits relative to the natural course of this most severe disease form, but also limits to treatment effectiveness, a case report suggests. While the child continues to show motor improvements…

Restoring levels of the SMN protein exclusively in nerve cells through gene therapy only partly eased symptoms and lengthened survival in a mouse model of severe spinal muscular atrophy (SMA), a study from France found. The gene therapy’s best results were observed when SMN levels were normalized systemically, or…