Scholar Rock recently announced that a poster presentation highlighting preclinical and Phase 1 healthy volunteer data for SRK-015 will be presented at the World Muscle Society Congress. The 24th Annual International Annual Congress of the World Muscle Society is being held in Copenhagen, Denmark this week. SRK-015 is a highly selective inhibitor of the activation […]
Biogen today announced that the journal Neuromuscular Disorders has published data from NURTURE, the first study investigating a treatment targeting the underlying cause of spinal muscular atrophy (SMA) in infants treated pre-symptomatically. Data from the NURTURE study demonstrated that infants who initiated treatment with SPINRAZA prior to the onset of clinical symptoms attained unparalleled results
Today, new data was presented demonstrating the ongoing benefit of risdiplam (RG7916) for the treatment of all types of spinal muscular atrophy (SMA). The data was presented at the 24th International Annual Congress of the World Muscle Society. Presentations include data from the FIREFISH, SUNFISH, and JEWELFISH clinical trials. The SMA program is a collaboration
Paul Guarino was ahead of his time when he started PG Sports as a Twitter page in 2011. Initially, the West Haven, CT native created a simple logo and shirts to promote the newly formed brand. As Guarino gained followers and interest grew in the shirts, he knew it was time for an upgrade. After
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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.
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
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
2019 Summit of Strength Program Continues its Success! Read More »
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
2019 SMA Researcher Meeting Summary: Clinical Drug Development Read More »
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
Cure SMA Newborn Screening Registry (NBSR) Read More »
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
Zolgensma Update and Approval Webinars Now Available Online Read More »
