[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, at-home one, as well as the scope of SMA issues and treatments. Here, we talk with the family of a young boy…

From candlelighting events to storytelling, efforts to mark Spinal Muscular Atrophy Awareness Month, observed each August, are underway to heighten awareness of the rare genetic disease and raise funds to fight it. Supporters are participating in a range of activities such as changing their social media profile photo, sharing…

[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 the chief of the SMA…

[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…

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…

Identifying those spinal muscular atrophy (SMA) patients who will benefit most from treatment with Zolgensma requires more than assessing traditional SMA types, according to a consensus statement from a panel of European experts. The statement covers 11 points, and highlights that clinical trials of Zolgensma to date involved a…

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…

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…

A readiness program to help clinical trial sites prepare to run spinal muscular atrophy (SMA) studies has been established by Cure SMA in collaboration with the pharmaceutical industry, other patient advocacy groups, and research institutes. The program is described in a position statement, “The SMA Clinical…