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…
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[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 fifth 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 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…
Michael Noon, 42, says the thing he hates most about spinal muscular atrophy (SMA) is having to constantly ask other people for assistance. “I’m keenly aware of the physical help I need, and as I’ve gotten weaker over the years, that’s always been my internal struggle,” he said. “You…
Zolgensma‘s approval as the first gene therapy for treating all forms of spinal muscular atrophy (SMA) in children up to 2 years old is another step forward — an exceptional and far-reaching one — in the seeming revolution underway in treating more diseases, and an increasing number of…
Within a lifetime, spinal muscular atrophy has leapt from a disease “nobody knew anything about” to a treatable — albeit chronic and still rare — condition. It is an “amazing” and welcome advance, say people who have lived with SMA for more than 20 years. “I’m kind of in…
