9 - 2019 - Cure SMA

AveXis Presents New Data at EPNS Continuing to Show Significant Therapeutic Benefit of Zolgensma® in Prolonging Event-free Survival Now up to 5 Years of Age in Patients with SMA Type 1

Leave a Comment / Front Page News, Research / Sarah McCall

AveXis, a Novartis company, recently announced that new interim data from the Phase 3 SPR1NT trial in pre-symptomatic patients as well as interim data from the ongoing Phase 3 STR1VE clinical program for Zolgensma showed positive outcomes, demonstrating age‑appropriate major milestone gain with pre‑symptomatic treatment and prolonged event-free survival* in patients with SMA Type 1. […]

AveXis Presents New Data at EPNS Continuing to Show Significant Therapeutic Benefit of Zolgensma® in Prolonging Event-free Survival Now up to 5 Years of Age in Patients with SMA Type 1 Read More »

Biogen Advances Spinal Muscular Atrophy (SMA) Clinical Research with New Study, DEVOTE, Evaluating a Higher Dose of SPINRAZA and Additional Data in a Broad Range of Patients

Leave a Comment / Clinical Trials, Research / Sarah McCall

Biogen today announced updates to the SPINRAZA (nusinersen) clinical development program including the initiation of a new global clinical trial, DEVOTE. The DEVOTE study will evaluate if a higher dose of SPINRAZA can provide even greater efficacy in the treatment of spinal muscular atrophy (SMA) across a broad patient population. In addition, new data further

Biogen Advances Spinal Muscular Atrophy (SMA) Clinical Research with New Study, DEVOTE, Evaluating a Higher Dose of SPINRAZA and Additional Data in a Broad Range of Patients Read More »

2019 Summit of Strength Program Continues its Success!

Leave a Comment / Events & Fundraising, Front Page News, Support & Care / Sarah McCall

The 2019 Cure SMA Summit of Strength Program has had a fantastic first half, with over 50 speakers sharing their expertise on numerous unique topics. We would like to thank these wonderful speakers for their participation, as well as all of the attendees for making this first half such a success. Summits are a single-day

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2019 SMA Researcher Meeting Summary: Clinical Drug Development

Leave a Comment / Uncategorized / Sarah McCall

The annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year, we had a record setting 735 attendees join together in Anaheim, CA. The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research. The meeting also furthers research

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Cure SMA Newborn Screening Registry (NBSR)

Leave a Comment / Front Page News, Support & Care / Sarah McCall

In 2018, Spinal Muscular Atrophy (SMA) was added to the federal Recommended Uniform Screening Panel (RUSP), a panel of diseases that newborns in the United States are recommended to be screened for. However, the decision on whether to implement newborn screening for a specific condition is up to each state. Since SMA’s inclusion on the

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Zolgensma Update and Approval Webinars Now Available Online

Leave a Comment / Front Page News, Research, Support & Care / Sarah McCall

A recording of last week’s webinar on Zolgensma updates is now available on Cure SMA’s YouTube channel. A presentation of the webinar can be found here. Panelists from the August 28th webinar included Dr. Peter Marks, M.D. Ph.D., Director, Center for Biologics Evaluation and Research (CBER) at FDA; Kenneth Hobby, President at Cure SMA; Mary

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