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June 24, 2019June 24, 2019

Anti-epileptic Keppra Could Be Candidate for Treatment of SMA, Study Finds

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The anti-epileptic medicine Keppra (levetiracetam) may have a neuroprotective effect for spinal muscular atrophy (SMA), according to a recent study. The study, “The Protective Effects of Levetiracetam on a Human iPSCs‑Derived Spinal ... Read more

June 21, 2019June 20, 2019

Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

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Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, ... Read more

June 21, 2019June 21, 2019

Small RNA Molecule miR-23a May Have Therapeutic Role in SMA, Study Says

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A small RNA molecule called miR-23a can prevent neuron degeneration and muscle wasting by acting as a protective modifier in cellular and animal models of spinal muscular atrophy (SMA), ... Read more

June 19, 2019June 19, 2019

Raising Levels of Alpha-COP Protein Seen to Ease Disease Severity in Mouse Model

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Elevated levels of the protein alpha-COP, involved in cellular transport and self-cleansing processes, extended survival in a mouse model of severe spinal muscular atrophy (SMA) without altering levels of the ... Read more

June 17, 2019June 17, 2019

‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

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People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most ... Read more

June 17, 2019June 17, 2019

Ontario SMA Patients Granted Wider Access to Spinraza

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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 ... Read more

June 14, 2019June 14, 2019

Zolgensma ‘Saved All of Our Lives’: A Family’s Journey Through Gene Therapy

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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 ... Read more

June 13, 2019June 13, 2019

European SMA Advocates Question EU’s Willingness to Reimburse for Zolgensma

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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, ... Read more

June 12, 2019June 21, 2019

SMA Candidate SRK-015 Shows Promise in Healthy Volunteers; Trial Ongoing

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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 ... Read more

June 10, 2019June 11, 2019

Expert Views on Likely Future Work into Zolgensma, Gene Therapies and Other SMA Treatments

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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, ... Read more

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