José Lopes, PhD,  managing science editor—

Monitoring liver function and immune reactions, being alert to signs of a respiratory infection, and making sure that a child’s vaccination schedule does not interfere with necessary corticosteroid use are among the key health and safety requirements for families and doctors interested in using Zolgensma to treat a young spinal muscular…

Ontario has expanded publicly funded access to Spinraza (nusinersen) to cover spinal muscular atrophy (SMA) types 2 and 3. The decision specifically covers presymptomatic patients with two or three copies of the SMN2 gene; patients with SMA for less than six months, two SMN2 copies, and…

Top-line results from a Phase 1 trial showed that SRK-015 had a positive safety profile and durable effects in healthy adult volunteers. These findings supported the start of the Phase 2 study TOPAZ (NCT03921528) to evaluate SRK-015’s efficacy to improve motor function of SMA patients. TOPAZ is currently…

[Editor’s note: This is the sixth and final entry in a series of articles on Zolgensma and treatments for SMA, the issues they raise, and possible discoveries to come, all drawn from recent interviews with neurologists and researchers involved in this work. Others in this series can be found…

[Editor’s note: This is the fourth in a series of articles on Zolgensma and treatments for SMA, the issues they raise, and possible discoveries to come, all drawn from recent interviews with neurologists and researchers involved in this work. Others in this series can be found here. SMA News…

[Editor’s note: This is the third in a series of articles on Zolgensma and treatments for SMA, the issues they raise, and possible discoveries to come, all drawn from recent interviews with neurologists and researchers involved in this work. Others in this series can be found here. SMA News…

[Editor’s note: This is the second in a series of articles on Zolgensma and treatments for SMA, the issues they raise, and possible discoveries to come, all drawn from recent interviews with neurologists and researchers involved in this work. Others in this series can be found here. SMA News…

[Editor’s note: This is the first in a series of articles on Zolgensma and treatments for SMA, the issues they raise, and possible discoveries to come, all drawn from recent interviews with neurologists and researchers involved in this work. Others in this series can be found here. SMA News…

The gene therapy Zolgensma (onasemnogene abeparvovec-xioi) has been newly approved to treat patients with spinal muscular atrophy (SMA) through intravenous (IV) delivery, meaning systemically. However, Novartis is also waiting on a more sweeping label, one allowing both intravenous and intrathecal (IT) delivery — that which is given via…

From its very first clinical trial — using  a dose widely frowned upon — Zolgensma has had a “transformative” impact on spinal muscular atrophy (SMA) and on gene therapy as a whole, said Jerry Mendell, MD, a principal investigator across Zolgensma’s clinical program. “We did our best, I think, to make…