Zolgensma Helps Babies Reach Age-Appropriate Motor Milestones, AveXis Reports
Babies with spinal muscular atrophy (SMA) treated with Zolgensma before experiencing symptoms continue to reach motor milestones in…
Articles by José Lopes, PhD
Higher Doses of Spinraza To Be Tested in New Phase 2/3 Trial
A new global Phase 2/3 clinical trial will test whether higher doses of Spinraza (nusinersen) provide greater efficacy…
Cure SMA Webinar Gives Update on Zolgensma Access, Insurance, and Data Concerns
With increasing insurance coverage and AveXis’ plans to broaden access to older spinal muscular atrophy (SMA) patients, the…
SMA STAT Genetic Test Offered Free in US to Enable Earlier Diagnosis
A new and faster genetic test for spinal muscular atrophy (SMA), called SMA STAT, will be offered free of…
Skye, First Baby in Connecticut Treated with Zolgensma, ‘Healthier and Stronger’
Skye, a nine-month-old baby with spinal muscular atrophy (SMA) type 1, is reaching new milestones every week and is…
EMA Grants Orphan Product Designation to Potential SMA Add-On Therapy Reldesemtiv
The European Medicines Agency (EMA) has granted orphan medicinal product designation to reldesemtiv, a potential add-on therapy for patients…
Additional Space for Zolgensma Production Goal of AveXis-Catalent Partnership
A new long-term partnership with Catalent Biologics will allow Zolgensma’s developer AveXis to increase production of the…
Risdiplam Raising SMN Levels in Older Patients in Ways That Seem Durable, Researcher Says of Early Trial Data
Treating teenagers and adults with oral risdiplam, a potential therapy for spinal muscular atrophy (SMA), led to sustained increases…
Spinraza European Label Updated to Reflect Long-term Benefits in Broad Range of SMA Patients
The European label for Spinraza (nusinersen) has been updated to include longer-term benefits seen in pre-symptomatic treatment…
Presymptomatic Spinraza Treatment Leads to SMA Children Reaching Otherwise Unachievable Milestones, Phase 2 Trial Shows
Presymptomatic infants treated with Spinraza (nusinersen) are able to hit motor milestones that are otherwise unachievable in the…