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…
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Tina and Torence Anderson’s sixth and youngest child, Malachi, came into the world on July 31, 2015. “When he was born, he was normal,” Tina said in a phone interview with SMA News Today. But eight weeks later,…
Now that the U.S. Food and Drug Administration has given its blessing to the AveXis gene therapy Zolgensma for children up to 2 years old with spinal muscular atrophy, the company hopes to win similar approval from the European Medicines Agency (EMA) later this year. AveXis,…
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 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…
